Bone Marrow Stem Cell Transplantation and Gene Therapy for Cystinosis
A presentation by Stephanie Cherqui, PhD, University of California, San Diego at the 2013 Day of Hope Cystinosis Research Foundation Family Conference, Balbo...
By: Natalieswish
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Bone Marrow Stem Cell Transplantation and Gene Therapy for Cystinosis - Video
A child born to an HIV-infected mother in Mississippi may be cured after a swiftly administered course of drugs. A number of factors make the child’s case unique, however, and clinicians caution that we have not discovered a general cure for HIV yet. Still, the medical first may hint at ways to fight the AIDS-causing virus .
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Editor’s Note: Excerpted from Shouting Won’t Help: Why I--and 50 Million Other Americans--Can’t Hear You , by Katherine Bouton, published by Sarah Crichton Books, an imprint of Farrar, Straus and Giroux, LLC. Copyright © 2013 Katherine Bouton. All rights reserved.
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http://rss.sciam.com/click.phdo?i=26ba9d76b5b91ce8e95b09bea6e3b5c4
A child born to an HIV-infected mother in Mississippi may be cured after a swiftly administered course of drugs. A number of factors make the child’s case unique, however, and clinicians caution that we have not discovered a general cure for HIV yet. Still, the medical first may hint at ways to fight the AIDS-causing virus .
Source:
http://rss.sciam.com/click.phdo?i=95337d9798ea17db628f12536ca0a05b
Editor’s Note: Excerpted from Shouting Won’t Help: Why I--and 50 Million Other Americans--Can’t Hear You , by Katherine Bouton, published by Sarah Crichton Books, an imprint of Farrar, Straus and Giroux, LLC. Copyright © 2013 Katherine Bouton. All rights reserved.
Source:
http://rss.sciam.com/click.phdo?i=26ba9d76b5b91ce8e95b09bea6e3b5c4
Muscle stiffness is a major clinical feature in Duchenne muscular dystrophy (DMD). DMD is the most common lethal inherited muscle-wasting disease in boys, and it is caused by the lack of the dystrophin protein. We recently showed that the extensor digitorum longus (EDL) muscle of mdx mice (a DMD mouse model) exhibits disease-associated muscle stiffness. Truncated micro- and mini-dystrophins are the leading candidates for DMD gene therapy. Unfortunately, it has never been clear whether these truncated genes can mitigate muscle stiffness. To address this question, we examined the passive properties of the EDL muscle in transgenic mdx mice that expressed a representative mini- or micro-gene (H2-R15, R2-15/R18-23/C, or R4-23/C). The passive properties were measured at the ages of 6 and 20 mo a...
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If cupid had studied neuroscience, he’d know to aim his arrows at the brain rather than the heart. Recent research suggests that for love to last, it’s best he dip those arrows in oxytocin. Although scientists have long known that this hormone is essential for monogamous rodents to stay true to their mates, and that it makes humans more trusting toward one another, they are now finding that it is also crucial to how we form and maintain romantic relationships.
http://rss.sciam.com/click.phdo?i=1e2c8b293a6e7840f25387be9dac539e
Muscle stiffness is a major clinical feature in Duchenne muscular dystrophy (DMD). DMD is the most common lethal inherited muscle-wasting disease in boys, and it is caused by the lack of the dystrophin protein. We recently showed that the extensor digitorum longus (EDL) muscle of mdx mice (a DMD mouse model) exhibits disease-associated muscle stiffness. Truncated micro- and mini-dystrophins are the leading candidates for DMD gene therapy. Unfortunately, it has never been clear whether these truncated genes can mitigate muscle stiffness. To address this question, we examined the passive properties of the EDL muscle in transgenic mdx mice that expressed a representative mini- or micro-gene (H2-R15, R2-15/R18-23/C, or R4-23/C). The passive properties were measured at the ages of 6 and 20 mo a...
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If cupid had studied neuroscience, he’d know to aim his arrows at the brain rather than the heart. Recent research suggests that for love to last, it’s best he dip those arrows in oxytocin. Although scientists have long known that this hormone is essential for monogamous rodents to stay true to their mates, and that it makes humans more trusting toward one another, they are now finding that it is also crucial to how we form and maintain romantic relationships.
http://rss.sciam.com/click.phdo?i=1e2c8b293a6e7840f25387be9dac539e
Potent growth-inhibitory effect of a dual cancer-specific oncolytic adenovirus expressing apoptin on prostate carcinoma.
Int J Oncol. 2013 Mar;42(3):1052-60
Authors: Zhang M, Wang J, Li C, Hu N, Wang K, Ji H, He D, Quan C, Li X, Jin N, Li Y
Abstract
Apoptin is a chicken anemia virus-derived, p53-independent, bcl-2-insensitive apoptotic protein with the ability to specifically induce apoptosis in various human tumor cells, but not in normal cells. To explore the use of apoptin in tumor gene therapy, we assessed a recombinant adenovirus expressing the apoptin protein (Ad-hTERTp-E1a-Apoptin) in order to determine its lethal and growth-inhibitory effects on PC-3 and RM-1 cells in vitro and its antitumor effect on solid tumors in vivo....
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Authors: Kurosaki T, Uematsu M, Shimoda K, Suzuma K, Nakai M, Nakamura T, Kitahara T, Kitaoka T, Sasaki H
Abstract
In this experiment, we developed anionic ternary complexes for effective and safe ocular gene delivery. Ternary complexes were constructed by coating plasmid DNA (pDNA)/polyethylenimine (PEI) complex with anionic polymers such as ?-polyglutamic acid (?-PGA) and chondroitin sulfate (CS). The cationic pDNA/PEI complex showed high gene expression on the human retinal pigment epithelial cell line, ARPE-19 cells. The pDNA/PEI complexes, however, also showed high cytotoxicity on the cells and aggregated strongly in the vitreous body. On the other hand, the anionic ternary complexes showed high gene expression on ARPE-19 cells without such cytotoxicity and aggregation. Afte...
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Authors: Kurosaki T, Uematsu M, Shimoda K, Suzuma K, Nakai M, Nakamura T, Kitahara T, Kitaoka T, Sasaki H
Abstract
In this experiment, we developed anionic ternary complexes for effective and safe ocular gene delivery. Ternary complexes were constructed by coating plasmid DNA (pDNA)/polyethylenimine (PEI) complex with anionic polymers such as ?-polyglutamic acid (?-PGA) and chondroitin sulfate (CS). The cationic pDNA/PEI complex showed high gene expression on the human retinal pigment epithelial cell line, ARPE-19 cells. The pDNA/PEI complexes, however, also showed high cytotoxicity on the cells and aggregated strongly in the vitreous body. On the other hand, the anionic ternary complexes showed high gene expression on ARPE-19 cells without such cytotoxicity and aggregation. Afte...
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Potent growth-inhibitory effect of a dual cancer-specific oncolytic adenovirus expressing apoptin on prostate carcinoma.
Int J Oncol. 2013 Mar;42(3):1052-60
Authors: Zhang M, Wang J, Li C, Hu N, Wang K, Ji H, He D, Quan C, Li X, Jin N, Li Y
Abstract
Apoptin is a chicken anemia virus-derived, p53-independent, bcl-2-insensitive apoptotic protein with the ability to specifically induce apoptosis in various human tumor cells, but not in normal cells. To explore the use of apoptin in tumor gene therapy, we assessed a recombinant adenovirus expressing the apoptin protein (Ad-hTERTp-E1a-Apoptin) in order to determine its lethal and growth-inhibitory effects on PC-3 and RM-1 cells in vitro and its antitumor effect on solid tumors in vivo....
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Research on human beings has expanded greatly due to progress and the evolution of society as well as customs. Not only the unceasing development of research on human beings, but also interference in the beginning and end of life with homologous and heterogonous human reproduction, surrogate motherhood, cloning, gene therapies, eugenics,euthanasia, dysthanasia, orthothanasia, assisted suicide, genetic engineering, reassignment surgery in cases of transsexuality, the use of recombinant DNA technology and embryonic stem cells, transplantation of human organs and tissues, biotechnology and many other scientific advances. Scientific progress goes faster than the real needs of human beings, who are the final recipient of the entire evolutionary progress. Hence, there is the need to scrutinize w...
MedWorm Sponsor Message: Find the best Christmas presents and January Sales in the UK with this simple shopping directory.
Research on human beings has expanded greatly due to progress and the evolution of society as well as customs. Not only the unceasing development of research on human beings, but also interference in the beginning and end of life with homologous and heterogonous human reproduction, surrogate motherhood, cloning, gene therapies, eugenics,euthanasia, dysthanasia, orthothanasia, assisted suicide, genetic engineering, reassignment surgery in cases of transsexuality, the use of recombinant DNA technology and embryonic stem cells, transplantation of human organs and tissues, biotechnology and many other scientific advances. Scientific progress goes faster than the real needs of human beings, who are the final recipient of the entire evolutionary progress. Hence, there is the need to scrutinize w...
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The Science Of The Next 150 Years: 50 Years in the Future [More]
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What scientific and technological milestones can we envision 50, 100 and 150 years hence?
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The Science Of The Next 150 Years: 50 Years in the Future [More]
http://rss.sciam.com/click.phdo?i=72f945621480bfc5969f6a7a29254aaf
What scientific and technological milestones can we envision 50, 100 and 150 years hence?
http://rss.sciam.com/click.phdo?i=bb22bbe61dfa476fb230c7787f0d7954
After the first description of a patient recognized as a MPS case was made in 1917, several similar cases were described and identified. Observations reported in the middle of the twentieth century concerning the presence of acid mucopolysaccharides (later called glycosaminoglycans, or GAGs) in tissues and especially in urine of patients were instrumental in providing an identity for these diseases, which became referred as "mucopolysaccharidoses" (MPS). In the late 1960's it was demonstrated that MPS were caused by defects in the breakdown of GAGs, and the specific enzyme deficiencies for the 11 types and subtypes of MPS were identified thereafter. Genes involved in the MPS were subsequently identified, and a large number of disease-causing mutations were identified in each one. Although ...
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