Category Archives: Gene therapy

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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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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WebMD News from HealthDay

By Amy Norton

HealthDay Reporter

THURSDAY, Feb. 21 (HealthDay News) -- When it comes to treating heart failure, the ultimate hope is to develop a therapy that repairs the damaged heart muscle.

Now, an early study hints at a way to do that by harnessing the body's natural capacity for repair.

Heart failure is a chronic, progressive condition where the heart cannot pump blood efficiently enough to meet the body's needs, which leads to problems like fatigue, breathlessness and swelling in the legs and feet. Most often, it arises after a heart attack leaves heart muscle damaged and scarred.

In the new study, researchers were able to use gene therapy to modestly improve symptoms in 17 patients with stage III heart failure -- where the disease is advanced enough that even routine daily tasks become difficult.

What is novel about the tactic, the researchers said, is that the gene therapy is designed to attract the body's own stem cells to the part of the heart muscle that's damaged. The hope is that the stem cells will then get some repair work done.

The findings, published Feb. 21 in the journal Circulation Research, are preliminary, and much more research needs to be done.

"This is a proof-of-concept study," explained lead researcher Dr. Marc Penn, a professor at Northeast Ohio Medical University in Rootstown, and director of research at Summa Cardiovascular Institute in Akron. But Penn and other heart failure experts said they were cautiously optimistic about the therapy's potential for at least some patients.

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Gene Therapy Shows Early Promise for Heart Failure

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.

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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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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...

Source:
http://www.medworm.com/index.php?rid=7067339&cid=c_449_68_f&fid=33708&url=http%3A%2F%2Fjap.physiology.org%2Fcgi%2Fcontent%2Fabstract%2F114%2F4%2F482%3Frss%3D1

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.

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QUEBEC, Feb. 4, 2013 /CNW Telbec/ - In the latest issue of the journal Molecular Therapy, Professor Jacques P. Tremblay (president of the Association of Gene Therapy of Quebec and researcher in the Research center of the Centre Hospitalier Universitaire (CHU) of Quebec) launches a call - with 50 other world experts in gene therapy - for the creation of an International Gene Therapy Consortium for Monogenic Diseases. The bases of this consortium will be established during a workshop, which will be held during the congress of the American Society of Gene and Cell Therapy (ASGCT) in Salt Lake City in May 2013.

Recent scientific progress in molecular biology and in genomics allowed during the recent years to identify the genes responsible for 10,000 hereditary diseases caused by a mutation in a single gene (for ex., Duchenne muscular dystrophy, Friedreich ataxia, haemophilia, etc.). On the other hand, recent progress of gene therapy resulted in treatments for some of these diseases previously considered incurable: hereditary immuno-deficiencies (the bubble children), a form of hereditary blindness (congenital amaurosis of Leber), etc. Also, for the first time, a gene-therapy treatment was approved for commercialization in Europe (for familial hyperchylomicronemia, a lipid disease). A Quebec team participated in the development of this treatment. The discovery of pluripotent stem cells, for which Dr. Yamanaka obtained the Nobel Prize in Medicine 2012, also allows to genetically correct the patient own cells and to differentiate them in various types of cells including those of heart and brain. These cells could then be re-transplanted to the patient without immunosuppression.

The research to develop treatments for these hereditary illnesses is at present made by small teams often financially supported by small patient associations. This fragmentation of the research and the sub-financing make more difficult the development of clinical trials. Professor Tremblay and his cosignatories indicate that with sufficient budgets, it would be possible to develop globally therapies for the most of these diseases during the next 2 decades.

The Regroupement qubcois des maladies orphelines (Quebec Coalition of Orphan Diseases) encourages initiatives that help develop treatments for rare genetic disorders.

The article that calls for the creation of the Consortium: Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases, Molecular Therapy, February 2013.

SOURCE: Regroupement qubcois des maladies orphelines

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Creation of an International Gene Therapy Consortium