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Fawn Creek township, Montgomery County, Kansas (KS) detailed profile

October 6th, 2024 2:38 am

Number of foreign born residents: 2 (6% naturalized citizens)

63% of Fawn Creek township residents lived in the same house 5 years ago.Out of people who lived in different houses, 62% lived in this county.Out of people who lived in different counties, 50% lived in Kansas.

Median price asked for vacant for-sale houses in 2000: $9,999

Median worth of mobile homes: $29,800

Housing units in Fawn Creek township with a mortgage: 181 (15 second mortgage, 16 home equity loan, 7 both second mortgage and home equity loan)Houses without a mortgage: 159

Year house built

Rooms in owner-occupied houses in Fawn Creek township, Kansas:

Rooms in renter-occupied apartments in Fawn Creek township, Kansas:

Bedrooms in owner-occupied houses and condos in Fawn Creek township:

Bedrooms in renter-occupied apartments in Fawn Creek township:

Cars and other vehicles available in Fawn Creek township in owner-occupied houses/condos:

Cars and other vehicles available in Fawn Creek township in renter-occupied apartments

Owners and renters by unit type in %

Breakdown of mean house values by ages of householders ($)

Most common industries for males (%):

Most common industries for females (%):

Most common occupations for males (%)

Most common occupations for females (%)

Most commonly used house heating fuel:

96.9% of residents of Fawn Creek township speak English at home.1.4% of residents speak Spanish at home (62% speak English very well, 38% speak English not well).0.6% of residents speak other Indo-European language at home (100% speak English very well).1.1% of residents speak Asian or Pacific Island language at home (100% speak English very well).0.1% of residents speak other language at home (100% speak English very well).

Size of family households: 294 2-persons, 167 3-persons, 73 4-persons, 75 5-persons, 12 6-persons, 9 7-or-more-persons,

Size of nonfamily households: 174 1-person, 21 2-persons, 6 3-persons,

391 married couples with children.57 single-parent households (11 men, 46 women).

Educational Attainment (%)

School Enrollment by Level of School (%)

Age and Sex of Sensory-Disabled Residents (Noninstitutionalized)

Age and Sex of Physically-Disabled Residents (Noninstitutionalized)

Age and Sex of Mentally-Disabled Residents (Noninstitutionalized)

Age and Sex of Self-Care Disabled Residents (Noninstitutionalized)

Age and Sex of Go-Outside-Home Disabled Residents (Noninstitutionalized)

Age and Sex of Residents with Employment Disability (Noninstitutionalized)

Year Householders Moved Into Unit in Fawn Creek township:

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Fawn Creek township, Montgomery County, Kansas (KS) detailed profile

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The Nanomedicine Revolution – PMC – National Center for Biotechnology …

October 6th, 2024 2:38 am

P T. 2012 Sep; 37(9): 512-517, 525.

Part 1: Emerging Concepts

The author is a Consultant Medical Writer living in New Jersey.

Nanoparticles may soon be used to transport diagnostic and therapeutic drugs to targeted sites not normally accessible, thereby improving treatment and reducing costs. Further research is still needed to establish the efficacy and safety of these nanomaterials.

This is the first in a series of three articles about nanomedicine. Part 2 will discuss the current and future clinical applications of nanomedicine. The third article in this series will focus on the regulatory and safety challenges presented by nanomedicine.

Nanomedicine, the application of nanotechnology to medicine, is currently at an early stage but it is expected to have a revolutionary impact on health care.1 Nanomedical research is heavily supported by public policy and investment, and is progressing rapidly.1,2 The continued development of nanomedicines has the potential to provide numerous benefits, including improved efficacy, bioavailability, doseresponse, targeting ability, personalization, and safety compared to conventional medicines.25 The most exciting concept in nanomedical research may be the design and development of multifunctional nanoparticle (NP) complexes that can simultaneously deliver diagnostic and therapeutic agents to targeted sites.5,6 These capabilities are unprecedented and represent tremendous progress toward improving patient diagnosis, treatment, and follow-up.6 However, despite these potential benefits, essential data regarding the pharmacokinetics, pharmacodynamics, and toxicity of many nanomaterials are currently lacking.5,7

Nanotechnology is a rapidly advancing field that is expected to have a revolutionary impact on many industries, including medicine.8,9 Nanotechnology has been made possible through the convergence of many scientific fields, including chemistry, biology, physics, mathematics, and engineering.1,2,9

A nanometer (nm) is one billionth of a meter, and the prefix nano- comes from the Greek word for dwarf.4,10 Nanotechnology provides scientists with new tools for the investigation, manipulation, and control of atoms, molecules, and submicroscopic objects, generally ranging from 1 to 100 nm.1,6 Nanotechnology allows scientists to take advantage of naturally occurring quantum effects at the nanoscale level that influence biological, physical, chemical, mechanical, and optical properties.6,10,11 These unique effects often give nanoscale materials desirable chemical, physical, and biological properties that differ from those of their larger, or bulk, counterparts.12

The convergence of nanotechnology and medicine has led to the interdisciplinary field of nanomedicine.6 Advances in genetics, proteomics, molecular and cellular biology, material science, and bioengineering have all contributed to this developing field, which deals with physiological processes on the nanoscale level.6,9 Many of the inner workings of a cell naturally occur on the nanoscale level, since the dimensions of many biologically significant molecules like water, glucose, antibodies, proteins, enzymes, receptors, and hemoglobin are already within the nanoscale range (see ).6,11 Many researchers are currently working on medical treatments, devices, and instruments that use nanotechnology to increase efficacy, safety, sensitivity, and personalization.11 Potentially beneficial properties of nanotherapeutics include improved bioavailability, reduced toxicity, greater dose response, and enhanced solubility compared with conventional medicines.2

This scale depicts the relative size of nanoscale, microscopic, and macroscopic objects. (Adapted from the National Cancer Institute.15)

The National Nanotechnology Initiative (NNI), a federal research and development program, defines nanotechnology as the science of materials and phenomena in the range of 1 to 100 nm in diameter.2,4,10 Many federal agencies, including the FDA and the Patent and Trademark Office (PTO), continue to use this definition.2 However, some experts say that this size limitation is artificial and misleading, since nanomaterials can have unique properties even in sizes up to several hundred nanometers.2

The National Institutes of Health (NIH) has presented an alternative definition of nanotechnology that doesnt rely on size; instead, it defines the field as (1) studies that use nanotechnology tools and concepts to study biology, (2) the engineering of biological molecules to have functions that differ from those that they have in nature, or (3) the manipulation of biological systems by methods more precise than standard molecular biological, synthetic, chemical, or biochemical approaches.2

Nanotechnology has the potential to be used in a wide range of products, including medicines, electronics, cosmetics, and foods.1,1315 According to the Project for Emerging Nanotechnologies at The Woodrow Wilson International Center for Scholars, more than 800 nanotechnology-based products are already on the market.9 Nanotechnology has been used in laptop computers, cell phones, digital cameras, water-filtration systems, and cosmetics.14,15 Nanotechnology research is also under way to improve the bioavailability of food nutrients and to develop food packaging that detects and prevents spoilage.14,16

Nanotechnology has also been applied to improve a number of medical products and processes;14,15 these include drugs, medical imaging, antimicrobial materials, medical devices, sunscreens, burn and wound dressings, dental-bonding agents, sunscreens, and protective coatings for eyeglasses.14,15 Nanotechnology has improved drug targeting and bioavailability, diagnostic imaging, biomarker detection sensitivity, and drug-delivery efficiency.16 Some nanomedicines that are currently on the market include doxorubicin HCl liposome injection (Doxil, Ortho Biotech) for ovarian cancer; daunorubicin citrate liposome injection (DaunoXome, Diatos) for advanced AIDS-related Kaposis sarcoma; and amphotericin B liposome injection (AmBisome, Gilead) for fungal infections.3,5 In addition, paints containing silver NPs, which have antimicrobial properties, are being used in indoor medical settings, such as in hospitals.17

Nanotechnology is a rapidly growing field. In 2008, nanotechnology was estimated to be a $10.5 billion industry in the U.S, mostly due to consumer product applications.17 It is estimated that the nanotechnology industry will grow to $1 trillion by 2015, representing an increase of about 100-fold in just 7 years.17

Nanomedicine has always been a major application for nanotechnology. 8 According to the National Science Foundation (NSF), by 2020, one-third of patents and start-up companies in the nanotechnology sector will involve biomedical applications. 8,18 The NSF also predicts that nearly half of future pharmaceuticals will have some nanotechnology components.4,18

The physical characteristics of NPs can differ in many ways that influence function.9 A discussion of several of these physical features follows.

NPs are inherently small, with at least one dimension in the range of 1 to 100 nm, although they can also be micrometer (m)-sized particles.6,9 NPs have novel structural, optical, and electronic properties that many larger molecules or bulk solids lack.9 They also have improved solubility, so they may be used to reinvestigate bulk drug counterparts that are known to have poor solubility.6 This property may provide the ability to convert insoluble or poorly soluble drugs into soluble aqueous suspensions, thus eliminating the need for toxic organic solvents.4 Another key benefit related to the small size of NPs is an increased bioavailability and circulation time.3 Studies have shown that particles under 200 nm have longer circulation times, compared with larger particles, irrespective of any surface modifications present.3

NPs come in a variety of shapes, including spheres, discs, hemispheres, cylinders, cones, tubes, and wires.6,9 NPs can also be hollow, porous, or solid.5 These characteristics of NPs can be selected on the basis of interactivity, loading capacity, and transport capabilities.6 For example, a hollow NP may be an attractive carrier for drug therapies or imaging contrast agents.6

One feature of NPs that gives them unique physical properties is a large surface area relative to size.2 As particle size decreases, total surface area increases exponentially ().2,11 An increase in surface area means that a greater proportion of atoms are located on the particle surface relative to the core.2 This phenomenon makes NPs more reactive compared with conventional larger molecules, or bulk solid counterparts.2 Increased surface area is also responsible for the enhanced water solubility and bioavailability that often occur with NPs.2

Illustration depicting the exponential increase in surface area that occurs with nanoscale materials. (Adapted from the National Technology Initiative.11)

The large surface area of NPs also allows them to be designed to include a broad range of surface characteristics, including conjugation with electrostatic charges or biomolecules.6 Such surface features can be strategically selected for targeting and other purposes and are therefore determined on that basis.9

If NPs are properly designed, their small size can enable them to cross physiological barriers to deliver drugs to sites that are not normally accessible by traditional means.6 For example, the increased permeability of an NP may allow it to transport cancer drugs into tumors by passing through neovessel pores that are less than 1 m in diameter.5 The increased permeability of NPs may also allow them to cross the bloodbrain barrier through the use of different uptake mechanisms.6

A wide variety of NPs and materials are used in nanomedicine, depending on the application.6 Among the most widely used are liposomes, polymers, quantum dots (QDs), iron oxide (IO) particles, and carbon nanotubes and nanoshells.6

A liposome is a spherical vesicle composed of a lipid bilayer membrane and an empty core that usually carries an aqueous solution.5 Liposomes are usually 90 to 150 nm in diameter and are thus slightly larger than conventional NPs.5 Liposomes are often designed to carry biomolecules (e.g., monoclonal antibodies, antigens) that are conjugated to the surface as ligands.5

Liposomes are often used in nanomedical research because they have many unique properties.5 The components of liposomes are similar to natural human cell membranes; thus, they confer liposomal drug delivery with several intrinsic benefits.5 Liposomes circulate in the bloodstream for an extended time, compared with non-liposomal drugs, providing a longer treatment effect. Liposomes also accumulate at the site of a tumor or infection, naturally locating and delivering higher drug levels to these targets.5 Liposomes can carry and deliver either hydrophilic or hydrophobic therapies, which can be stored in their empty cores.6 By using lipids of different fatty-acid-chain lengths, scientists can construct liposomes to be temperature-sensitive or pH-sensitive, thereby permitting the controlled release of their contents only when they are exposed to specific environmental conditions.5

In contrast to other materials, data on the safety and efficacy of many polymers already exist; therefore, polymer NPs are widely used in nanomedical research.3 Polymer NPs can be fabricated in a wide range of varieties and sizes, ranging from 10 nm to 1 m.3,5 Some polymer NPs can facilitate drug release for weeks and do not accumulate in the body.3,5,6 As such, polymeric NPs are considered promising carriers for numerous medications, including those used in cancer, cardiovascular disease, and diabetes treatments; bone-healing therapies; and vaccinations.3 Contrast agents can also be conjugated to the surface of polymeric NPs, allowing them to be used in diagnostic imaging.5

Biodegradable polymers are of particular interest, since they can be fully metabolized and removed from the body.6Poly-lactic-co-glycolic acid (PLGA) is an especially intriguing example of a biodegradable polymer, since relative proportions of polylactic acid (PLA) and polyglycolic acid (PGA) can be used to fine-tune the biodegradability of PLGA.6

Quantum dots (QDs) are semiconductor nanocrystals that range in size from 2 to 10 nm and usually consist of 10 to 50 atoms.4,5 Although QDs have been used in electronics and optics for 20 years, they have only recently been applied to nanomedical research.5 The most commonly used QDs for biomedical applications contain cadmium selenide (CdSe) or cadmium telluride (CdTe).4 QDs containing indium phosphide (InP) and indium arsenide (InAs) are also frequently used.4

QDs have unique optical and electronic properties, making them valuable as luminescent probes and giving them tremendous potential in many biomedical applications.4,5 QDs are intrinsically fluorescent and emit light over a broad range, from the near-ultraviolet (UV) to mid-infrared spectrum.9 They have size-dependent optical properties, extraordinary photostability, and surface properties that can be fine-tuned, which make them ideal for optical imaging.4 QDs have molar extinction coefficients that are 10 to 50 times larger than those of organic dyes, making them much brighter in in vivo conditions.5 They have long blood circulation times and can fluoresce for several months in vivo.5

QDs also have sufficient surface area to attach agents for simultaneous targeted drug delivery and in vivo imaging or for tissue engineering.4 Many uses of QDs for in vivo imaging have already been reported, including lymph node and angiogenic vessel mapping and cell subtype isolation.5 QDs are very efficient agents for cancer diagnosis in vivo, because the extremely small size of the QDs allows unimpeded access to systemic circulation and surface modifications can target them to neoplastic sites.4 Additional potential uses for QDs include image-guided surgery, light-activated therapies, and diagnostic tests.19

Surface coatings have been found to enhance the surface fine-tunability and increase the fluorescent yield of QDs.4 They may also reduce the adverse effects that can be elicited by QDs containing Cd, Se, and As, which are toxic materials.4 At present, the investigation of QDs is restricted to in vitro and animal studies because of toxicity concerns regarding these heavy metals.5,19 Novel methods to produce new generations of QDs in which toxic materials are reduced or absent are being pursued for future applications in humans.5

Superparamagnetic NPs, like iron oxide (SPIO) and magnetite, have been used for years as nontargeted contrast agents for magnetic resonance imaging (MRI).1,5,17 However, these NPs do have superparamagnetic properties that allow them to be directed in situ with the use of a magnetic field.17 They also have a long retention time in circulation, are usually biodegradable, and have low toxicity.5 They are therefore excellent candidates for producing imageable therapeutic nanodevices.5

In addition to possessing other desirable properties, SPIO NPs can also be functionalized (designed) to achieve specific tumor targeting.5 SPIO NPs are increasingly being used for the development of target-specific MRI contrast agents.5 To date, SPIO NPs have been used for many applications, such as the delivery of antibiotics and drugs with simultaneous enhancement of MRI contrast and for the separation of bacteria from biomolecules.17

Carbon nanotubes are composed of a distinct molecular form of carbon atoms that give them unusual thermal, mechanical, and electrical properties.5 For example, they are 100 times stronger than six times their weight in steel.5 Carbon nanotubes modified with polyethylene glycol (PEG) are surprisingly stable in vivo, with long circulation times and low uptake by the reticuloendothelial system (RES).5 Carbon nanotubes have been used for the delivery of imaging and therapeutic agents and in the transport of DNA molecules into cells.5 The nanoscale dimensions of single-walled and multiwalled carbon nanotubes, along with their electrocatalytic properties and high surface area, have compelled researchers to utilize them as nanoelectrodes.20

Carbon nanoshells are composed of a silica core that is covered by a thin metallic shell, usually composed of gold.5 Carbon nanoshells have an ability to scatter light, a feature that is useful for cancer imaging.5 However, their primary use continues to be in thermal ablation therapy.5 Alternatively, focused lasers have been useful for cancer thermotherapy, but they cannot discriminate between diseased and healthy tissue.1 However, when carbon nanoshells are used for targeting in thermal ablation therapy, thermal energy passes through healthy tissue without causing harm, killing only the targeted tumor cells.5 In mice, carbon nanoshells and near-infrared spectroscopy (NIRS) thermal ablation therapy completely eliminated colon carcinoma cell tumors in vivo.5

The aforementioned, and other, NPs are used to construct multifunctional NP complexes that mix and match different features, or functionalizations, in order to achieve an intended purpose.17 A multifunctional NP complex may be designed to include the following components ():3,5,21

surface ligands that target the attachment of NPs to specific locations (e.g., organs, cells, or tissues).

linker molecules that release the cargo carried by the NP at the target site in response to a remote trigger or specific environmental cues.

a core that encapsulates targeting or imaging cargo or has optical or magnetic properties (gold, SPIO) that can localize the NP at the target site.

one or more therapeutic or diagnostic cargoes that are encapsulated in the NP core or attached to its surface.

a coating, such as PEG, that improves biocompatibility and/or enhances bioavailability by increasing circulation times and slowing clearance from the body.

Diagram representing a multifunctional NP complex. The carrier particle, payload, and surface modifiers can be customized. PEG = polyethylene glycol. (Adapted from Ferrari M. Nat Rev Cancer 2005;5[3]:161171.21)

One of the most interesting capabilities in nanomedicine is the functionalization of NPs.7 Functionalization involves altering properties of an NP through chemical or physical modifications that are applied to achieve a desired effect.7 This process can provide local or directed delivery, prolong drug effects, facilitate transport into target cells, locate a tumor or area of infection, provide feedback regarding efficacy or drug delivery, or reduce blood flow shear effects.9 A discussion of the various approaches to functionalizing NPs follows.

NPs can be administered locally or can be actively targeted using cell-specific ligands, magnetic localization, and/or size-based selectivity.3 Many factors need to be considered when constructing targeted NPs, including size, biocompatibility, target affinity, avoidance of the RES, and stability in the blood, as well as the ability to facilitate controlled drug release.7

Magnetic polymer nanocomposites or magnetoliposomes grafted with drug molecules have great potential for targeted drug delivery.3 These NPs have potentially favorable biodistribution and pharmacokinetic profiles, which can be enhanced by the external application of a static magnetic field at the site of action.3 For example, in one study, MRI confirmed that magnetic NPs had migrated toward neodymium/iron/boron (NdFeB) magnets that had been placed outside the peritoneal cavity, above grafts of a human ovarian carcinoma.3

NPs can be engineered to incorporate a wide variety of chemotherapeutic agents that can be targeted directly and specifically to the tumor site for better efficacy and safety.4 NPs can also be filled with contrast agents for imaging purposes.6 In comparison to small-molecule contrast agents, multifunctional NP complexes or NPs used in diagnostic imaging have the advantage of a large surface area that allows targeting through surface modifications and the ability to simultaneously deliver therapeutic agents.1

One way in which NPs can be functionalized for specific applications is through surface conjugation.17 Nanoparticle surfaces can be conjugated with a wide range of diagnostic or therapeutic agents.1 Some candidate biomolecules for NP surface conjugation are cell-penetrating peptides (CPPs) that enhance intracellular delivery, fluorescent dyes for imaging, and agents for genetic therapy such as small inhibitory RNA (siRNA).7 Nanoparticle surfaces, conjugated with a targeting molecule that binds to highly expressed tumor cell receptors, can also facilitate the transport of imaging contrast agents that provide increased sensitivity and specificity, which aid in tumor detection.5

The surfaces of NPs can also be conjugated with drug therapies. 3 Surface conjugation with ligands that specifically bind to the target site can enhance the efficacy of NP drug-delivery systems while significantly reducing toxicity.4 In cancer treatment, tumor targeting can be achieved by conjugating a molecule or biomarker (such as a peptide, protein, or nucleic acid) that is known to bind to tumor cell receptors on the NP surface.5

NPs are generally cleared from circulation by immune system proteins called opsonins, which activate the immune complement system and mark the NPs for destruction by macrophages and other phagocytes.3 Neutral NPs are opsonized to a lesser extent than charged particles, and hydrophobic particles are cleared from circulation faster than hydrophilic particles.3 NPs can therefore be designed to be neutral or conjugated with hydrophilic polymers (such as PEG) to prolong circulation time.3 The bioavailability of liposomal NPs can also be increased by functionalizing them with a PEG coating in order to avoid uptake by the RES.5 Liposomes functionalized in this way are called stealth liposomes.5

NPs are often covered with a PEG coating as a general means of preventing opsonization, reducing RES uptake, enhancing biocompatibility, and/or increasing circulation time.5 SPIO NPs can also be made water-soluble if they are coated with a hydrophilic polymer (such as PEG or dextran), or they can be made amphophilic or hydrophobic if they are coated with aliphatic surfactants or liposomes to produce magnetoliposomes. 5 Lipid coatings can also improve the biocompatibility of other particles.3

NPs can also be designed so that they can be activated to release therapeutic or diagnostic cargo in response to a site-specific or remote trigger.3 Properties that can be used to functionalize NPs for controlled release include pH, temperature, magnetic field, enzymatic activity, or other features such as light or radiofrequency signals.6 NPs constructed with pH-responsive materials can be designed to trigger drug release at a target site upon detecting a change in pH.3 For example, the mildly acidic environment inside inflammatory and tumor tissues (pH 6.8) and cellular vesicles, such as endosomes (pH 5.56.0) and lysosomes (pH 4.55.0), can be exploited to trigger drug release.3

Thermally responsive linkers, consisting of nucleic acids, peptides, proteins, lipids, carbohydrates, or polymers, can also be used to attach one or more agents for controlled release from the NP.3 When the thermally responsive linker is exposed to a specific temperature or temperature range (the trigger temperature), the linker is disrupted and the agent is released.3 For example, DNA molecules with heat-labile hydrogen bonding between complementary strands can act as a heat-sensitive linker.5 An NP can also be designed to include several thermally responsive linkers that are designed to disrupt at different temperatures, enabling drug delivery to occur in a specific order over varied periods of time.3

The release of agents from NPs can also be achieved through the incorporation of bonds that degrade under other specific conditions at the target site.5 For example, tumor-specific processes may be exploited to break a bond and trigger the release of a therapeutic agent.5 Tumor site-specific conditions that could be used to trigger release might include abnormal oxygen levels, unique biomarkers, or exposure to proteolytic enzymes that are overexpressed in tumors.5

The tunability of NP properties is an important and powerful concept.20 NPs have a broad range of tunable biologic, optical, magnetic, electric, and mechanical features that differ dramatically from the same materials in larger forms because of modified quantum mechanics occurring at the nanoscale level.6 By changing the size of an NP, researchers can fine-tune many different properties of nanomaterials.11 For example, they can achieve different colors of fluorescence by changing the size of an NP, allowing a means of color coding or labeling during diagnostic imaging applications.11

Nanomedicines might someday provide answers to longstanding problems in medical research, ranging from poor drug solubility to a lack of target specificity for therapeutic compounds.2 Nanomedicine also has tremendous promise as a noninvasive tool for diagnostic imaging, tumor detection, and drug delivery because of the unique optical, magnetic, and structural properties of NPs that other tools do not possess.1

Nanomedicine presents new opportunities to improve the safety and efficacy of conventional therapeutics.5 Drugs with low bioavailability can now be targeted directly to the site required.3,5 The large surface area and greater reactivity of NPs may allow dose reduction of a drug, which can improve toxicity profiles and patient compliance.2,3 The large surface area of NPs can also increase the dissolution rate, saturation solubility, and intracellular uptake of drugs, improving in vivo performance.2,3 Combining encapsulation, release modalities, and surface modifications to improve therapeutic targeting or bioavailability could improve the efficacy of NP formulations several-fold compared with bulk counterparts.4 Targeted NPs can also transport large doses of therapeutic agents into malignant cells while sparing normal, healthy cells.4,5

One of the most exciting applications of nanomedicine is the use of multifunctional NP complexes for simultaneous non-invasive targeting, imaging, and treatment.1,4,5 Multifunctional NPs for cancer treatment can potentially include a variety of tumor targeting ligands as well as imaging and therapeutic agents that allow noninvasive monitoring and treatment.5 Multifunctional NPs that include fluorescent dyes can also provide in vivo imaging of biologic events during drug administration as well as potential diagnostic labels for the early detection and localization of tumors.7

Recent research efforts are also focused on developing magnetic NPs for the targeted delivery of various therapeutic or diagnostic agents.4 Interest in magnetic NP targeting applications is inspired by the possibility of detecting the particles by MRI and then correlating the results with histologic findings after treatment.3 Polymer/SPIO composites are the most common NPs used for theranostics (diagnostics).3 More than one cancer drug can also be incorporated on a polymer/IO conjugate backbone.3 The drugs can be released at the tumor site, allowing them to act together synergistically, potentially achieving higher efficacy.3 Because SPIO NPs generate heat when exposed to an alternating field, electromagnetic fields can also be applied externally for remote activation of SPIO NPs for thermal ablation therapy.5

Nanotechnologies have already transformed genetic and biological analysis through devices that examine molecular biomarkers.1 Compared with conventional modalities, these tests can be conducted more rapidly, reliably, and cost effectively via in vitro and in vivo diagnostic technologies that, for example, might use nanochips or QDs.1 Nanotechnologies can also produce diagnostic devices that are more sensitive and can detect earlier signs of metabolic imbalances, which can assist in the prevention of diseases like diabetes and obesity.20 The continued application of nanotechnologies to produce better and more cost-effective means of detecting molecular biomarkers will also open the way to the more routine practice of personalized medicine.1

Despite the benefits that nanomedicine has to offer, much research is still required to evaluate the safety and toxicity associated with many NPs.3 Much of nanomedical research has concentrated on drug delivery, with relatively few studies focusing on the pharmacokinetics or toxicity of NPs.7 Investigating NP pharmacokinetics, pharmacodynamics, and potential long-term toxicity in vivo is essential to monitoring the effects of NPs on patient populations.5 Validating every nanotherapeutic agent for safety and efficacy, whether drug, device, biologic, or combination product, presents an enormous challenge for researchers and the FDA, which is currently struggling to formulate testing criteria and accumulate safety data.2,3

Studies are also needed to assess the immunogenicity of NPs.20 Nanotherapeutics and diagnostics may present unexpected toxic effects because of increased reactivity compared with their bulk counterparts.2 The most frequently reported side effect after injection of a nanotherapeutic agent seems to be a hypersensitivity reaction, which may be caused by activation of the immune complement system.6 The main molecular mechanism for in vivo NP toxicity is thought to be the induction of oxidative stress through the formation of free radicals.3 In excess, free radicals can cause damage to lipids, protein, DNA, and other biological components through oxidation. Several authors have reported that intrinsic characteristics of NPs, such as aspect ratio and surface area, can be pro-oxidant and pro-inflammatory.7 However, the formation of free radicals in response to an NP can also have other causes, such as the reaction of phagocytic cells to foreign material, insufficient antioxidants, the presence of transition metals, environmental factors, and other intrinsic chemical or physical properties.3

Research to evaluate the size and surface properties of NPs may also help to identify the critical dimensions at which they tend to significantly accumulate in the body.20 NPs have an increased ability to cross biological barriers and therefore have the potential to accumulate in tissues and cells because of their small size.2 The possible tissue accumulation, storage, and slow clearance of these potentially free radicalproducing particles, as well as the prevalence of numerous phagocytes in the RES, may make organs such as the liver and spleen the main targets of oxidative stress.3,6

This lack of data about potential toxicity issues forces nanomedical research to focus predominantly on polymer NPs, for which safety and efficacy data already exist.3 In fact, several nanomedicines containing polymer NPs are already approved by FDA.3 Unlike other materials that may become toxic in NP form, the lipid NPs are also considered to be biocompatible and tolerable.3 Consequently, biodegradable, soluble, nontoxic NPs, such as polymers, liposomes, and IO particles, are much more desirable to use in nanomedicines than biopersistent components are.5 The use of NPs like carbon nanotubes, QDs, and some metallic nanocarriers that are not biodegradable might be more problematic.1,7 This characteristic need not discourage nanomedical research with these NPs but should reinforce efforts to identify additional biodegradable shapes, materials, and surface treatments.7

Although nanomedicine is still at an early stage of development, several drugs that utilize nanotechnology have been approved and marketed, and many others are being studied.2,3 Nanomedicines potentially offer a means of earlier diagnosis; more effective, safer, and personalized treatments; as well as reduced health care costs.1 Many experts agree that nanomedicine will create a paradigm shift that revolutionizes health care within the next 10 years.2,8 However, for significant progress to be made toward this goal, much more work is needed to establish testing criteria, validate efficacy, and accumulate safety data for various nanotherapeutic agents and materials.2,3

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The Nanomedicine Revolution - PMC - National Center for Biotechnology ...

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Fawn Creek, Montgomery County, Kansas Population and Demographics

October 6th, 2024 2:38 am

In this article, we'll explore the population statistics for Fawn Creek, Kansas, including popular demographics data like median age, number of households, household income, gender, employment and unemployment rates, occupations, religion, and more.

We are using the latest American Community Survey (ACS) 5-Year survey data from the US Census Bureau, which is accurate up to 2021.

There is a lot of data that lets us see how many people live in Fawn Creek, Kansas. The most basic data is the total population, which is the total number of people living in Fawn Creek, Kansas. The estimated population of Fawn Creek, Kansas is 1,618 people, with a median age of 46.5.

We can look at the total population in Fawn Creek over the last 5 years to see how much it has changed.

1,618

1,888

+8.19%

1,745

1,853

+10.36%

1,679

We can also look at how the population has changed over the last 5 years by each of the race/ethnicity types as defined by the US Census Bureau.

This data can be very important for a number of reasons, including social and economic research, planning and development, and marketing to a multi-cultural population.

Note that the Native group includes both Native American and Alaskan Native people.

The age and gender of a population is another interesting demographic statistic because it lets us track trends in the population over time. For example, we can see how the population has changed over the last 5 years by median age as a total, and also by male and female.

The median age of Fawn Creek, Kansas gives you an idea of the age distribution, with half of the population being older than the median age and half being younger.

This can then be used to infer and compare against birth rates, parent ages, and more metrics to understand the population. For example, an increasing median age indicates an aging population, which can be a sign of a declining population in terms of birth rates and workforce participation.

The latest median household income of Fawn Creek, Kansas is $58,992.00.

In simple terms, the median income is the middle income of a group of people. Half of the people in the group make more than the median income, and half make less. The median income is a good indicator of the overall income of a group of people, and can be used to compare against other metrics such as the average income, per capita income, and more.

We can also look at the median income by age to see how the median income varies by age, and how it compares to the overall median income for Fawn Creek.

Under 25

$21,667.00

25 to 44

$65,714.00

45 to 64

$77,969.00

65 and over

$37,500.00

Whenever we ask what is the average household income in Fawn Creek, we are actually talking about the mean household income.

This is calculated by adding up all the incomes of all the households in Fawn Creek, and then dividing that number by the total number of households. This is a good way to get a general idea of the average income of a group of people, but it can be skewed by a very high or very low incomes.

The average household income of Fawn Creek, Kansas is currently $71,206.00.

In terms of accurately summarizing income at a geographic level, the median income is a better metric than the average income because it isn't affected by a small number of very high or very low incomes.

If you had an area where the average income was greater than the median, it can mean that there is significant income inequality, with income being concentrated in a small number of wealthy households.

4.51% of households in Fawn Creek are classed as high income households (making $200,000+ per year).

The US Census Bureau divides households into income tiers based on the median income for the area. This is a good way to compare the income of Fawn Creek against other areas.

Less than $24,999

26.78%

$25,000 to $49,999

15.28%

$50,000 to $74,999

19.94%

$75,000 to $99,999

17.03%

$100,000 to $149,999

8.15%

$150,000 to $199,999

8.30%

$200,000 or more

4.51%

The per capita income in Fawn Creek is $32,441.00.

Per capita income is the average income of a person in a given area. It is calculated by dividing the total income of Fawn Creek by the total population of Fawn Creek.

This is different from the average or mean income because it includes and accounts for all people in Fawn Creek, Kansas, including people like children, the elderly, unemployed people, retired people, and more.

We can also look at the education levels in Fawn Creek to see how many people have a high school degree, a bachelor's degree, or a graduate degree.

Educational attainment is a good indicator of the overall education level of a population, and can be used to compare against other metrics such as the average income, per capita income, and more to see how education levels affect income, unemployment rates, and more.

Master's degree or higher

4.89%

Bachelor's degree

18.85%

Some college or associate's degree

9.93%

High school diploma or equivalent

65.61%

Less than high school diploma

0.72%

Employment rates are all based around the total population in Fawn Creek that are over the age of 16.

The total population of Fawn Creek over the age of 16 is 1,375.

Of those people, a total of 60.70% are working or actively looking for work. This is called the labor force participation rate.

The participation rate is a useful market measure because it shows the relative amount of labor resources available to the economy.

The employment to total population rate in Fawn Creek is 56.40%.

We can look at the employment rates by age to see how it compares to the overall employment rate.

Another very interesting employment statistic we can look at is the employment and unemployment rates by race in Fawn Creek. The table below shows the rates for each of the ethnicity groups types defined by the US Census Bureau.

In this section we can look at the most common occupations in Fawn Creek as well as the gender breakdown and earnings of them.

The total population of civilian employees that are 16 years old or older in Fawn Creek is 775, with median earnings of $38,750.00.

Women in Fawn Creek, Kansas earning approximately 66.00% of the men's earnings.

In the table below, we can break down the population and earnings even further by occupation. The list of occupation categories below will show you how many people are employed in each category and the median earnings of each profession.

The table below shows the same occupations from the list above, but we have split them by male and female to see how many male and females work in each occupation, the median earnings, and the male to female ratio of earnings.

That last metric is important because it can be used to look at the gender pay gap between men and women.

A household defined bu the US Census Bureau is a group of people who occupy a housing unit. A housing unit is a house, apartment, mobile home, group of rooms, or single room occupied as separate living quarters.

There are currently 687 households in Fawn Creek, with an average household size of 2.36 people.

A family is defined as a group of two or more people related by birth, marriage, or adoption who live together in the same household.

There are 490 families in Fawn Creek with an average family size of 2.68 people.

The four categories of household by marital status are:

The table below shows the total number of households and families, with the average sizes of each.

Of the 490 families in Fawn Creek, 15.30% are considered to be below the poverty threshold.

The table below shows the latest poverty thresholds in Fawn Creek:

There are 764 housing units in Fawn Creek, Kansas.

The table below shows the split between occupied and vacant units:

4.84% of the total 764 housing units in Fawn Creek are rental units. This is approximately 37 properties.

For owner-occupied housing units, a total of 615 are occupied by the owner - or 80.50% of the total.

The median rent for a property in Fawn Creek is $1,079.00.

In the chart below, we can look at the number of rental properties in Fawn Creek that fall into a particular rent range. These can then be used to compare with other areas, or to see how they have changed over time.

No rent paid

35

Less than $500

0

$500 to $999

10

$1,000 to $1,499

27

$1,500 to $1,999

Excerpt from:
Fawn Creek, Montgomery County, Kansas Population and Demographics

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An Introduction to Nanomedicine – AZoNano

October 6th, 2024 2:38 am

An Introduction to Nanomedicine  AZoNano

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An Introduction to Nanomedicine - AZoNano

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Nanomedicine Market is expected to show growth from 2024 to 2030, reported by Maximize Market Research – openPR

October 6th, 2024 2:38 am

Nanomedicine Market is expected to show growth from 2024 to 2030, reported by Maximize Market Research  openPR

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Nanomedicine Market is expected to show growth from 2024 to 2030, reported by Maximize Market Research - openPR

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Gene therapy: advances, challenges and perspectives – PMC

October 6th, 2024 2:37 am

ABSTRACT

The ability to make site-specific modifications to the human genome has been an objective in medicine since the recognition of the gene as the basic unit of heredity. Thus, gene therapy is understood as the ability of genetic improvement through the correction of altered (mutated) genes or site-specific modifications that target therapeutic treatment. This therapy became possible through the advances of genetics and bioengineering that enabled manipulating vectors for delivery of extrachromosomal material to target cells. One of the main focuses of this technique is the optimization of delivery vehicles (vectors) that are mostly plasmids, nanostructured or viruses. The viruses are more often investigated due to their excellence of invading cells and inserting their genetic material. However, there is great concern regarding exacerbated immune responses and genome manipulation, especially in germ line cells. In vivo studies in in somatic cell showed satisfactory results with approved protocols in clinical trials. These trials have been conducted in the United States, Europe, Australia and China. Recent biotechnological advances, such as induced pluripotent stem cells in patients with liver diseases, chimeric antigen receptor T-cell immunotherapy, and genomic editing by CRISPR/Cas9, are addressed in this review.

Keywords: Gene therapy, Genetic Vectors, Gene transfer, horizontal, CRISPR-Cas9, CAR-T cell, Genetic therapy, Clustered regularly interspaced short palindromic repeats

A habilidade de fazer modificaes pontuais no genoma humano tem sido o objetivo da medicina desde o conhecimento do DNA como unidade bsica da hereditariedade. Entende-se terapia gnica como a capacidade do melhoramento gentico por meio da correo de genes alterados (mutados) ou modificaes stio-especficas, que tenham como alvo o tratamento teraputico. Este tipo de procedimento tornou-se possvel por conta dos avanos da gentica e da bioengenharia, que permitiram a manipulao de vetores para a entrega do material extracromossomal em clulas-alvo. Um dos principais focos desta tcnica a otimizao dos veculos de entrega (vetores) que, em sua maioria, so plasmdeos, nanoestruturados ou vrus sendo estes ltimos os mais estudados, devido sua excelncia em invadir as clulas e inserir seu material gentico. No entanto, existe grande preocupao referente s respostas imunes exacerbadas e manipulao do genoma, principalmente em linhagens germinativas. Estudos em clulas somticas in vivo apresentaram resultados satisfatrios, e j existem protocolos aprovados para uso clnico. Os principais trials tm sido conduzidos nos Estados Unidos, Europa, Austrlia e China. Recentes avanos biotecnolgicos empregados para o aprimoramento da terapia gnica, como clulas-tronco pluripotentes induzidas em pacientes portadores de doenas hepticas, imunoterapia com clulas T do receptor do antgeno quimera e edio genmica pelos sistema CRISPR/Cas9, so abordados nesta reviso.

Keywords: Terapia gnica, Vetores genticos, Transferncia gentica horizontal, CRISPR-Cas9, CAR-T cell, Terapia gentica, Repeties palindrmicas curtas agrupadas e regularmente espaadas

In 1991, James Watson declared that many people say they are worried about the changes in our genetic instructions. But these (genetic instructions) are merely a product of evolution, shaped so we can adapt to certain conditions which might no longer exist. We all know how imperfect we are. Why not become a little better apt to survive?.(1) Since the beginning, humans understand that the peculiar characteristics of the parents can be transmitted to their descendents. The first speculation originated from the ancient Greek students, and some of these theories continued for many centuries. Genetic-scientific studies initiated in the early 1850s, when the Austrian monk, Gregor Mendel, in a series of experiments with green peas, described the inheritance pattern by observing the traces that were inherited as separate units, which we know today as genes. Up until 1950, little was known as to the physical nature of genes, which was when the American biochemist, James Watson, and the British biophysicist, Francis Crick, developed the revolutionary model of the double strand DNA. In 1970, researchers discovered a series of enzymes that enabled the separation of the genes in predetermined sites along the DNA molecule and their reinsertion in a reproducible manner. These genetic advances prepared the scenario for the emergence of genetic engineering with the production of new drugs and antibodies, and as of 1980, gene therapy has been incorporated by scientists.(2,3)

In this review, we cover gene therapy, the different methodologies of genetic engineering used for this technique, its limitations, applications, and perspectives.

The ability to make local modificiations in the human genome has been the objective of Medicine since the knowledge of DNA as the basic unit of heredity. Gene therapy is understood as the capacity for gene improvement by means of the correction of altered (mutated) genes or site-specific modifications that have therapeutic treatment as target. Further on, diffrent strategies are described, which are often used for this purpose.(4)

Currently, gene therapy is an area that exists predominantly in research laboratories, and its application is still experimental.(5) Most trials are conducted in the United States, Europe, and Australia. The approach is broad, with potential treatment of diseases caused by recessive gene disorders (cystic fibrosis, hemophilia, muscular dystrophy, and sickle cell anemia), acquired genetic diseases such as cancer, and certain viral infections, such as AIDS.(3,6)

One of the most often used techniques consists of recombinant DNA technology, in which the gene of interest or healthy gene is inserted into a vector, which can be a plasmidial, nanoestrutured, or viral; the latter is the most often used due to its efficiency in invading cells and introducing its genetic material. On , a few gene therapy protocols are summarized, approved and published for clinical use, exemplifying the disease, the target, and the type of vector used.(3)

Gene therapy protocols

Although several protocols have been successful, the gene therapy process remains complex, and many techniques need new developments. The specific body cells that need treatment should be identified and accessible. A way to effectively distribute the gene copies to the cells must be available, and the diseases and their strict genetic bonds need to be completely understood.(3) There is also the important issue of the target cell type of gene therapy that currently is subdivided into two large groups: gene therapy of the germline(7) and gene therapy of somatic cells.(8) In germline gene therapy, the stem cells, e.g., with the sperm and egg, are modified by the introduction of functional genes, which are integrated into the genome. The modifications are hereditary and pass on to subsequent generations. In theory, this approach should be highly effective in the fight against genetic and hereditary diseases. Somatic cell gene therapy is when therapeutic genes are transferred to a patients somatic cells. Any modification and any effects are restricted only to that patient and are not inherited by future generations.

In gene therapy, a normal gene is inserted into the genome to replace an abnormal gene responsible for causing a certain disease. Of the various challenges involved in the process, one of the most significant is the difficulty in releasing the gene into the stem cell. Thus, a molecular carrier called a vector is used to release the gene, which needs to be very specific, display efficiency in the release of one or more genes of the sizes necessary for clinical applications, not be recognized by the immune system, and be purified in large quantities and high concentrations so that it can be produced and made available on a large scale. Once the vector is inserted into the patient, it cannot induce allergic reactions or inflammatory process; it should increase the normal functions, correct deficiencies, or inhibit deleterious activities. Furthermore, it should be safe not only for the patient, but also for the environment and for the professionals who manipulate it. Finally, the vector should be capable to express the gene, in general, for the patients entire life.(3,9)

Although the efficacy of viral vectors is confirmed, recently some studies demonstrated that the use of these carriers presented with several limitations. The presence of viral genetic material in the plasmid is a strong aggravating factor, since it can induce an acute immune response, besides a possible oncogenic transformation. Currently, there are two main approaches for genetic modifications of the cells, namely: virus-mediated () and via physical mechanisms, from preparations obtained by advanced nanotechnology techniques.(5) Within this context, included are polymers that form networks that capture a gene and release its load when they penetrate the cells, such as DNA microinjections,(10) cationic polymers,(11) cationic liposomes,(12,13) and particle bombardment.(14)

Viral vectors for gene therapy

Each exogenous material introduction technique differs from the other and depends on the type of application proposed. Some are more efficient, others more apt to carry large genes (>10kB) and integrate with the genome, allowing a permanent expression.(1)

Hematopoietic stem cells have become ideal targets for gene transfer due to the high potential for longevity and the capacity for self-renovation. One example of this combination of gene therapy and stem cells would be the production of gene transfer vectors for the creation of induced pluripotent stem cells (iPS), in order to generate the differentiation of the iPS and afford an additional phenotype from this differentiated derived cell. Patients with chronic liver disease and infection by the hepatitis virus (e.g., hepatitis B virus and hepatitis C virus), which require a liver transplant, may be likely to undergo the hepatic transplantation of mature hepatocytes or those derived from iPS.(15) Not only the transfer of genes might be needed to convert stem cells into hepatocytes; since the transplanted cells are susceptible to reinfection by the hepatitis virus, the transfer of a vector that encodes a short hairpin RNA directed against the virus would provide the transferred cells with resistance or immunity to reinfection. Resistant cells can repopulate the liver over time and restore normal hepatic function ().(15)

Combination of stem cells and gene therapy

shRA: short hairpin RNA; iPS: induced pluripotent stem cells.

Chimeric antigen recipient T (CAR-T) cell therapy is a type of immunotherapy that involves manipulation/reprogramming of immune cells (T lymphocytes) of the patients themselves, in order to recognize and attack the tumor T cells. Initial advancement in the design of the first CAR generation, by Eshhar et al.,(16) was marked by the fusion of a single chain fragment variable (scFv) to a transmembrane domain and an intracellular signaling unit: chain CD3 zeta.(17,18) This design combined the active element of a well-characterized monoclonal antibody with a signaling domain, increasing the recognition of the tumor-specific epitope and the activation of T cells, without depending on molecules from the histocompatibility complex.

An improvement in the first generation of CAR was made by means of integrating co-stimulating molecules necessary for signal transduction. The stimulatory recipient most commonly used in this CAR generation is CD28. This recipient acts as a second activating event of the route, enabling a marked proliferation of T cells along with an increased expression of cytokines.(19)

The most recent generation of CAR incorporated the addition of a co-stimulatory domain addition to increase the CAR function. Co-stimulatory molecules as recipients of the tumor necrosis factor (CD134 or CD137) are required for this methodology. In summary, the most recent forms of CAR include scFv, the initial chain of CD3-, along with the stimulatory chains of CD28 and CD134 or CD137.(20)

With the third CAR generation, Zhong et al., demonstrated an improvement in T cell activation of the Akt route (protein kinase B), which regulates the cell cycle. According to other studies, this last generation shows greater persistence of the T cells in comparison with the second generation of CAR.(21)

The most critical point of the adverse effects of CAR-T therapy is the identification of non-tumor cells that express the target epitope by CAR. Tumor antigens are molecules highly expressed in the tumor cells, but are not exclusive of these cells. For example, the CD19 antigen can be found in normal or malignant B cells, and the CAR design for the CD19 target in not capable of distinguishing them.(20,22) Other common toxicity for CAR-T therapy (and many other types of immunotherapy for cancer) is the cytokine release syndrome (CRS). Activation of the immune system after CAR-T infusion can induce a rapid increase in the levels of inflammatory cytokines.(20,23)

New developments in the design of vectors and trials with CAR-T provide balance and reinforcement in safety for amplification of the clinical application. The progressive improvement in the CAR trials has already advanced, as was observed from the first to the third generation. Knowledge and experience acquired in the assessment of CAR-T toxicity will increase the success of the progressive improvements for future trials.

During the 1980s, in the genome of Escherichia coli, a region was identified with an uncommon pattern, in which a highly variable sequence was intercalated by a repeated sequence with no known function. In 2005, it was assumed that the variable sequences were of extra-chromosomal origin, acting as an immune memory against phages and plasmids, starting the then unknown CRISPR system (Clustered Regularly Interspaced Short Palindromic Repeats) and Cas (Associated Proteins), that shines since 2012 as one of the primary biotechnological tools for gene edition.(24) Originating in the immune-adaptive system of procaryontes, this mechanism recognizes the invading genetic mateiral, cleaves it into small fragments, and integrates it into its own DNA. In a second infection by the same agent, the following sequence occurs: transcription of the CRISPR locus, RNAm processing, and creation of small fragments of RNA (crRNAs) that form complexes with the Cas proteins, and these recognize the alien nucleic acids and finally destroy them.(24)

Based on this natural mechanism, the CRIPSR technique was developed enabling editing of the target-specific DNA sequences of the genome of any organism by means of basically three molecules: nuclease (Cas9), responsible for cleavage of the double-strand DNA; an RNA guide, which guides the complex to the target; and the target DNA, as is shown in .(25,26)

CRISPR Cas-9 system. The technique involves basically three molecules: one nuclease (generally wild type Cas-9 of Streptococcus pyogenes), an RNA guide (known as single guide RNA), and the target (frequently the DNA)

Due to its simplicity and its precision when compared to other techniques (Zinc-Finger Nucleases, TALENs, and Gene Targeting), the CRISPR system arrives as a versitile tool that promotes the genetic editing by means of inactivation (knockout gene KO), integration of exogenous sequences (knock-in), and allele substitution, among others.(27,28)

The guide RNA hybridizes with the target DNA. Cas-9 recognizes this complex and should mediate cleavage of the DNA double strand and reparation in the presence of a (homologous) donor DNA. The result of this process is the integration of an exogenous sequence into the genome (knock-in) or allele substitution.

The rapid advancement of this new technology allowed the performance of translational trials in human somatic cells, using genetic editing by CRISPR. The first applications with a therapeutic focus already stood out in describing even the optimization steps of the delivery systems and specificity for the safety and efectiveness of the system.(28,29)

Researchers from the University of California and of Utah recently were successful in correcting the mutation of the hemoglobin gene, which originates sickle cell anemia. CD34+ cells from patients who are carriers of sickle cell anemia were isolated, edited by CRISPR-Cas9, and after 16 weeks, the results showed a reduction in the expression levels of the mutated gene and an increased gene expression of the wild type.(29)

The technology referred to is in use mainly in monogenic genetic pathologies, which, despite being rare, can reach about 10 thousand diseases already described.(4) Phase 1 clinical trials are foreseen for 2017, as well as the appearance of companies geared toward the clinical use of this system.

The possibility of genetically modifying germlines has been the object of heated discussion in the field of science for a long time. Bioethics is always present when new techniques are created, in order to assess the risks of the procedure and the moral implications involved.

A large part of the scientific community approves genetic therapy in somatic cells, especially in cases of severe disorders, such as cystic fibrosis and Duchenne muscular dystrophy.

In 2015, however, Chinese researchers went beyond the moral issues and announced, for the first time, the genetic modification of embryonic cells using the CRISPR-Cas9 technique. Next, another Chinese group also reported the conduction of the same process done with the intention of conferring resistance to HIV by insertion of the CCR5 gene mutation. The genetic analysis showed that 4 of the 26 embryos were successfully modified. The result clearly reveals the need for improving the technique, alerting that, possibly, such trials could be previously tested in animal models.(4,30)

These recent publications rekindled the debate regarding genetic editing. On one side, the Japanese Ethics Committee declared that the manner in which the experiment was conducted was correct, since there had been approval by the local Ethics Committee for the study conducted, as well as the consent of the egg donors. In the United Kingdom, the first project for healthy human embryo editing was approved. On the other hand, American research groups remained conservative, reiterating their position of not supporting this type of experiment and declaring that they await improvement in the techniques and of the definitions of ethical issues.(30)

Since the declaration of James Watson in 1991, in reference to the likely optimization of human genetics, gene therapy has advanced throughout the decades, whether by optimization of the types of vectors, by the introduction of new techniques, such as induced pluripotent stem cells in combination with current models of genetic editing (CRISPR-Cas9), and even by trials in germ cells, bringing with it the contradictory ethical and moral aspects that accompany the technique.

Local successes have already solidified the viability of treatments using gene therapy in clinical practice, as an alternative form for patients with congenital diseases or monogenic disorders and cancer, especially when the pharmacological or surgical interventions do not show good results.

The design of new experimental vectors, the increase in efficiency, the specificity of the delivery systems, and the greater understanding of the inflammatory response induction may balance the improvement of safety with the expansion of techniques in clinical applications. Yet the knowledge and experience acquired with the careful assessment of toxicity of these technologies also allow significant advances in the application of these methods.

Therefore, historically, gene therapy and the discovery of antibiotics and chemotherapy agents, or any new technology, need more clarifying preclinical studies. In the future, there is the promise of applying these techniques in several fields of Medicine and a greater percentage of clinical trials.

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Gene therapy: advances, challenges and perspectives - PMC

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Meeting on the Mesa to Highlight Cell and Gene Therapy Opportunities, Challenges – BioSpace

October 6th, 2024 2:37 am

Meeting on the Mesa to Highlight Cell and Gene Therapy Opportunities, Challenges  BioSpace

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Meeting on the Mesa to Highlight Cell and Gene Therapy Opportunities, Challenges - BioSpace

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Ferring opens doors to Finnish manufacturing hub as supply of its bladder cancer gene therapy continues to grow – FiercePharma

October 6th, 2024 2:37 am

Ferring opens doors to Finnish manufacturing hub as supply of its bladder cancer gene therapy continues to grow  FiercePharma

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Ferring opens doors to Finnish manufacturing hub as supply of its bladder cancer gene therapy continues to grow - FiercePharma

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Meet Boston’s National STEM Champion who’s a junior in high school studying gene therapy – CBS Boston

October 6th, 2024 2:37 am

Meet Boston's National STEM Champion who's a junior in high school studying gene therapy  CBS Boston

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Meet Boston's National STEM Champion who's a junior in high school studying gene therapy - CBS Boston

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Gene therapy research offers hope for kids with life-altering condition – WCVB Boston

October 6th, 2024 2:37 am

Gene therapy research offers hope for kids with life-altering condition  WCVB Boston

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Gene therapy research offers hope for kids with life-altering condition - WCVB Boston

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Is gene therapy the next big step in vision loss treatment? – Medical News Today

October 6th, 2024 2:37 am

Is gene therapy the next big step in vision loss treatment?  Medical News Today

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Is gene therapy the next big step in vision loss treatment? - Medical News Today

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Protein’s Role in Insulin Signaling Could Have Implications for Gene Therapy – AJMC.com Managed Markets Network

October 6th, 2024 2:37 am

Protein's Role in Insulin Signaling Could Have Implications for Gene Therapy  AJMC.com Managed Markets Network

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Protein's Role in Insulin Signaling Could Have Implications for Gene Therapy - AJMC.com Managed Markets Network

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Scientists overcome major challenge in gene therapy and drug delivery – News-Medical.Net

October 6th, 2024 2:37 am

Scientists overcome major challenge in gene therapy and drug delivery  News-Medical.Net

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Scientists overcome major challenge in gene therapy and drug delivery - News-Medical.Net

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Innovative gene therapy for hemophilia – healthcare-in-europe.com

October 6th, 2024 2:37 am

Innovative gene therapy for hemophilia  healthcare-in-europe.com

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Innovative gene therapy for hemophilia - healthcare-in-europe.com

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The Largest Network of Research Sites Vetted to Execute Complexities of Cell & Gene Therapy (CGT) Trials Now Includes 1,500 Sites – PR Newswire

October 6th, 2024 2:37 am

The Largest Network of Research Sites Vetted to Execute Complexities of Cell & Gene Therapy (CGT) Trials Now Includes 1,500 Sites  PR Newswire

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The Largest Network of Research Sites Vetted to Execute Complexities of Cell & Gene Therapy (CGT) Trials Now Includes 1,500 Sites - PR Newswire

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Cell therapy weekly: Promising Phase I results for Parkinsons disease cell therapy – RegMedNet

October 6th, 2024 2:37 am

Cell therapy weekly: Promising Phase I results for Parkinsons disease cell therapy  RegMedNet

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Cell therapy weekly: Promising Phase I results for Parkinsons disease cell therapy - RegMedNet

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Weight loss drug breakthroughs, gene therapies, and more: 8 clinical trials to watch right now – Quartz

October 6th, 2024 2:37 am

Weight loss drug breakthroughs, gene therapies, and more: 8 clinical trials to watch right now  Quartz

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Weight loss drug breakthroughs, gene therapies, and more: 8 clinical trials to watch right now - Quartz

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Targeting CREB-binding protein (CBP) abrogates colorectal cancer stemness through epigenetic regulation of C-MYC – Nature.com

October 6th, 2024 2:37 am

Targeting CREB-binding protein (CBP) abrogates colorectal cancer stemness through epigenetic regulation of C-MYC  Nature.com

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Targeting CREB-binding protein (CBP) abrogates colorectal cancer stemness through epigenetic regulation of C-MYC - Nature.com

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Forge Biologics Announces the FUEL AAV Manufacturing Platform to Provide Developers with a More Efficient Solution for Gene Therapy Production -…

October 6th, 2024 2:37 am

Forge Biologics Announces the FUEL AAV Manufacturing Platform to Provide Developers with a More Efficient Solution for Gene Therapy Production  Business Wire

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Forge Biologics Announces the FUEL AAV Manufacturing Platform to Provide Developers with a More Efficient Solution for Gene Therapy Production -...

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Ninth Circuit Decision Marks Critical Legal Victory for U.S. FDA in Mission to Protect Patients from Unregulated Cell Therapy Products – PR Newswire

October 6th, 2024 2:37 am

Ninth Circuit Decision Marks Critical Legal Victory for U.S. FDA in Mission to Protect Patients from Unregulated Cell Therapy Products  PR Newswire

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Ninth Circuit Decision Marks Critical Legal Victory for U.S. FDA in Mission to Protect Patients from Unregulated Cell Therapy Products - PR Newswire

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