Gene therapy, also called gene transfer therapy, introduction of a normal gene into an individuals genome in order to repair a mutation that causes a genetic disease. When a normal gene is inserted into the nucleus of a mutant cell, the gene most likely will integrate into a chromosomal site different from the defective allele; although that may repair the mutation, a new mutation may result if the normal gene integrates into another functional gene. If the normal gene replaces the mutant allele, there is a chance that the transformed cells will proliferate and produce enough normal gene product for the entire body to be restored to the undiseased phenotype.
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cancer: Gene therapy
Knowledge about the genetic defects that lead to cancer suggests that cancer can be treated by fixing those altered genes. One strategy
Human gene therapy has been attempted on somatic (body) cells for diseases such as cystic fibrosis, adenosine deaminase deficiency, familial hypercholesterolemia, cancer, and severe combined immunodeficiency (SCID) syndrome. Somatic cells cured by gene therapy may reverse the symptoms of disease in the treated individual, but the modification is not passed on to the next generation. Germline gene therapy aims to place corrected cells inside the germ line (e.g., cells of the ovary or testis). If that is achieved, those cells will undergo meiosis and provide a normal gametic contribution to the next generation. Germline gene therapy has been achieved experimentally in animals but not in humans.
Scientists have also explored the possibility of combining gene therapy with stem cell therapy. In a preliminary test of that approach, scientists collected skin cells from a patient with alpha-1 antitrypsin deficiency (an inherited disorder associated with certain types of lung and liver disease), reprogrammed the cells into stem cells, corrected the causative gene mutation, and then stimulated the cells to mature into liver cells. The reprogrammed, genetically corrected cells functioned normally.
Prerequisites for gene therapy include finding the best delivery system (often a virus, typically referred to as a viral vector) for the gene, demonstrating that the transferred gene can express itself in the host cell, and establishing that the procedure is safe. Few clinical trials of gene therapy in humans have satisfied all those conditions, often because the delivery system fails to reach cells or the genes are not expressed by cells. Improved gene therapy systems are being developed by using nanotechnology. A promising application of that research involves packaging genes into nanoparticles that are targeted to cancer cells, thereby killing cancer cells specifically and leaving healthy cells unharmed.
Some aspects of gene therapy, including genetic manipulation and selection, research on embryonic tissue, and experimentation on human subjects, have aroused ethical controversy and safety concerns. Some objections to gene therapy are based on the view that humans should not play God and interfere in the natural order. On the other hand, others have argued that genetic engineering may be justified where it is consistent with the purposes of God as creator. Some critics are particularly concerned about the safety of germline gene therapy, because any harm caused by such treatment could be passed to successive generations. Benefits, however, would also be passed on indefinitely. There also has been concern that the use of somatic gene therapy may affect germ cells.
Although the successful use of somatic gene therapy has been reported, clinical trials have revealed risks. In 1999 American teenager Jesse Gelsinger died after having taken part in a gene therapy trial. In 2000 researchers in France announced that they had successfully used gene therapy to treat infants who suffered from X-linked SCID (XSCID; an inherited disorder that affects males). The researchers treated 11 patients, two of whom later developed a leukemia-like illness. Those outcomes highlight the difficulties foreseen in the use of viral vectors in somatic gene therapy. Although the viruses that are used as vectors are disabled so that they cannot replicate, patients may suffer an immune response.
Another concern associated with gene therapy is that it represents a form of eugenics, which aims to improve future generations through the selection of desired traits. Some have argued that gene therapy is eugenic but that it is a treatment that can be adopted to avoid disability. To others, such a view of gene therapy legitimates the so-called medical model of disability (in which disability is seen as an individual problem to be fixed with medicine) and raises peoples hopes for new treatments that may never materialize.
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Gene therapy | medicine | Encyclopedia Britannica
- Governor Hochul Celebrates the Opening Of New York's First Cell and Gene Therapy Hub at Roswell Park Comprehensive Cancer Center in Buffalo - PR Web - December 19th, 2024
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- Gov. Hochul celebrates opening of first cell and gene therapy hub in NYS - WIVB.com - News 4 - December 19th, 2024
- Muscular Dystrophy Association and Coalition to Cure - GlobeNewswire - December 19th, 2024
- Atsena Therapeutics Announces Dosing Completed in Part A of - GlobeNewswire - December 19th, 2024
- 'A milestone moment': Roswell Park celebrates opening New York's first cell and gene therapy hub - WKBW 7 News Buffalo - December 19th, 2024
- Gene therapy to prevent stillbirth and premature delivery developed - News-Medical.Net - December 19th, 2024
- Breaking through the blood-brain barrier - Science - December 19th, 2024
- Cell therapy weekly: partnerships for advancing cell and gene therapies - RegMedNet - December 19th, 2024
- Roswell Park Opens Cell, Gene Therapy Hub - WGRZ.com - December 19th, 2024
- Cartherics gets $300k grant to advance Cell and Gene Therapy development - ETHealthWorld - December 19th, 2024
- ELMCRx Solutions Offers Cell & Gene Therapy Support Through Partnership with Emerging Therapy Solutions (ETS) - Business Wire - December 19th, 2024
- Fueling the Future of Gene Therapies with Manufacturing Innovation, Upcoming Webinar Hosted by Xtalks - PR Web - December 19th, 2024
- Concinnity secures 3M Seed funding to advance AI-driven gene therapy safety - Tech.eu - December 19th, 2024
- Viral Vectors-Based Gene Therapy for Non-Human Primates Market to Reach Over USD 92.76 Million by 2034 - EIN News - December 19th, 2024
- The pharma industry's silence on RFK Jr., and efforts by parents to develop gene therapies for their children - STAT - December 19th, 2024
- Tenaya reports positive early data on heart gene therapy - Investing.com - December 19th, 2024
- Unraveling The Complexity Of Cell Therapy: Advancements And Challenges - Life Science Leader Magazine - November 27th, 2024
- Novartis wagers more than $1B on gene therapies for the nervous system - BioPharma Dive - November 27th, 2024
- Gene therapy for geographic atrophy in age-related macular degeneration: current insights - Nature.com - November 27th, 2024
- Novartis buys gene therapy startup Kate Therapeutics, joining pursuit of muscular dystrophy treatment - STAT - November 27th, 2024
- At MGB's gene therapy institute, effort to win first venture capital investments continues - The Business Journals - November 27th, 2024
- Neurogene reports death of Rett patient left in critical condition by high dose of gene therapy - Fierce Biotech - November 27th, 2024
- Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy - CGTLive - November 27th, 2024
- Why This Gene-Therapy Companys Stock Is Rising 228% - Yahoo! Voices - November 27th, 2024
- How Minaris is Tackling the Scalability Challenge in Cell and Gene Therapy: A Conversation with CEO, Dr. Hiroto Bando - geneonline - November 27th, 2024
- RNA editing is the next frontier in gene therapy heres what you need to know - The Conversation - November 27th, 2024
- Assessment of gene therapy viral vectors in RPE cells - News-Medical.Net - November 27th, 2024
- Retinal organoids and RPE models for retinal gene therapy development - News-Medical.Net - November 27th, 2024
- China Vows to Bolster Gene Therapy Research in Key Biotech Hub - Bloomberg - November 27th, 2024
- Gene Therapy - Volume 31 Issue 11-12, November 2024 - Nature.com - November 27th, 2024
- Iovance Biotherapeutics Announces the Promotion of Raj Puri, M.D., Ph.D. to Chief Regulatory Officer - GlobeNewswire - November 27th, 2024
- Patient Dies in Gene Therapy Trial, But FDA Permits Neurogene to Proceed With Low Dose - MedCity News - November 27th, 2024
- New CRISPR system pauses genes, rather than turning them off permanently - Livescience.com - November 27th, 2024
- Liver-targeting gene therapy lowers mice whole-body SMA symptoms - SMA News Today - November 27th, 2024
- Bright breakthroughs: Real stories of beating rare disease - Science - November 27th, 2024
- Sarepta Therapeutics Announces Global Licensing and Collaboration Agreement with Arrowhead Pharmaceuticals for Multiple Clinical and Preclinical siRNA... - November 27th, 2024
- A Year of DMD Gene Therapy Trial Failures - AJMC.com Managed Markets Network - November 3rd, 2024
- Hemophilia B: Gene Therapy Shows Promise - Medscape - November 3rd, 2024
- Around the Helix: Cell and Gene Therapy Company Updates October 30, 2024 - CGTLive - November 3rd, 2024
- 2024 PharmaVoice 100s: Cell and Gene Therapy Pioneers - PharmaVoice - November 3rd, 2024
- Cell therapy weekly: support for commercialization of complex therapies - RegMedNet - November 3rd, 2024
- Lexeo shares early data on Alzheimers gene therapy - Endpoints News - November 3rd, 2024
- Medicaid Aiming to Improve Patient Access to High-Cost Therapies - AJMC.com Managed Markets Network - November 3rd, 2024
- The Significance of Gene Therapy in Neuromuscular Medicine at the 2025 MDA Conference: Paul Melmeyer, MPP - Neurology Live - November 3rd, 2024
- OHSU researchers identify gene that could be key to future HIV vaccine - OHSU News - November 3rd, 2024
- Purespring gene therapy reduces kidney scarring in mice and is stably expressed in pigs - Fierce Biotech - November 3rd, 2024
- Data Roundup: October 2024 Features Update for TCR-Based Autologous Cell Therapy in Melanoma, the First Clinical Demonstration of Therapeutic RNA... - November 3rd, 2024
- NewBiologix Launches Xcell to Accelerate, Optimize, and Scale Gene and Cell Therapy Production - Business Wire - November 3rd, 2024
- Vertex Pharmaceuticals and CRISPR Therapeutics Casgevy: the 200 Best Inventions of 2024 - TIME - November 3rd, 2024
- Addressing gene and cell therapy commercialization challenges - TechTarget - November 3rd, 2024
- University of Pennsylvania gene therapy spinout Interius BioTherapeutics doses patient, achieves CAR therapy first - The Business Journals - November 3rd, 2024
- Roche will aim to tackle gene therapy challenges through Dyno deal - The Pharma Letter - November 3rd, 2024
- Behind the Breakthroughs: How to Turn $1,000,000 CAR Ts into Real Medicines - Inside Precision Medicine - November 3rd, 2024
- Terumo automates manufacturing to expand cell & gene therapies - European Pharmaceutical Manufacturer - November 3rd, 2024
- 12-Year-Old Leaves Washington DC Hospital As The First Patient To Receive Approved Gene Therapy For Sickle Cell Disease - AfroTech - November 3rd, 2024
- Lexeo Therapeutics Announces Positive Interim Data for - GlobeNewswire - November 3rd, 2024
- New FDA designations granted to NCATS for rare disease therapies. - NCBI - October 22nd, 2024
- $1.8 Million Awarded to Study the Durability of Gene Therapy - University of Arkansas Newswire - October 22nd, 2024
- By the numbers: US leads charge of cell and gene therapies - BioWorld Online - October 22nd, 2024
- University of Arkansas Researcher Awarded $1.8M for Gene Therapy Study - Arkansas Business - October 22nd, 2024
- Cellectis to Present Data on TALE-Base Editors and Non-Viral Gene Therapy at the ESGCT 31st Annual Congress - StockTitan - October 22nd, 2024
- Around the Helix: Cell and Gene Therapy Company Updates October 16, 2024 - CGTLive - October 22nd, 2024
- Japan mulls ways to boost cell, gene therapy approvals - BioWorld Online - October 22nd, 2024
- A New Type of Gene Therapy Shows Promise for Treating Retinitis Pigmentosa - Managed Healthcare Executive - October 22nd, 2024
- Buy, Sell, Hold: Cell and Gene Therapy - Part 2 - BioPharm International - October 22nd, 2024
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- Buy, Sell, Hold: Cell and Gene Therapy - Part 3 - Pharmaceutical Technology Magazine - October 22nd, 2024
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- Cellectis to Present Data on TALE-Base Editors and Non-Viral Gene Therapy at the ESGCT 31st Annual Congress - The Manila Times - October 22nd, 2024
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