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From Genetics to Gene Therapy: Molecular Pathology of Human Disease (UCL Molecular Genetics)

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  1. 2. Nonviral Vectors for Gene Therapy, Part 1: v. 53, Pt. 1 (Advances in Genetics)
  2. 3. Gene Therapy and Gene Delivery Systems (Advances in Biochemical Engineering/biotechnology)
  3. 4. A Model for Gene Therapy
  4. 5. Gene Therapy for Neurological Disorders
  5. 6. The Ethics of Human Gene Therapy

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Gene Therapy



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Gene Therapy Part I: HIV, the New Silver Bullet

In a ground-breaking experiment, French scientists have stripped HIV of its bad traits and used it to deliver apparently lasting and reliable gene therapy. Where do we go from here? Post from: Notes From the Lizard Lair Gene Therapy Part I: HIV, the New Silver Bullet

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Gene Therapy Stalls Development Of Huntington's Disease In Mice

Researchers at the California Institute of Technology have shown that a highly specific intrabody (an antibody fragment that works against a target inside a cell) is capable of stalling the development of Huntington's disease in a variety of mouse models. "Gene therapy in these models successfully attenuated the symptoms of Huntington's disease and increased life span," notes...

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Gene Therapy Halts Brain Disease in Two Boys

Researchers have used a modified AIDS virus to halt a devastating brain disease in two young boys. The treatment, in which the virus delivered a therapeutic gene, marks the first time gene therapy has been successfully used against X-linked adrenoleukodystrophy (ALD)–a disorder that is always fatal if untreated. With this proof of principle, scientists hope [...]

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Researchers to figure out tricky viruses, adapt for gene therapy

11/9/2009 11/9/2009 11/8/2009 11/8/2009 11/7/2009 11/6/2009 11/6/2009 11/5/2009 11/5/2009 11/4/2009 11/4/2009 11/4/2009 11/3/2009 11/3/2009 11/3/2009 Researchers to figure out tricky viruses, adapt for gene therapy (11/9/2009)Tags:, Viruses have an uncanny ability to trick cells into letting them inside. Once inside, they take over, says Brian Bothner, a faculty member in Montana State...

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Gene therapy success in severe brain disorder applauded by the STOP ALD Foundation

First gene therapy success in boys with fatal brain disorder -- the Stop ALD Foundation, having spurred a successful European gene therapy trial, is now pressing to bring this therapy to the US. The foundation was started by families with children who have died or suffered from adrenoleukodystrophy, the disease highlighted in the movie Lorenzo's Oil. A report of...

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Gene therapy beats back brain wasting disease

A breakthrough mix of stem cell and gene therapy halted a lethal brain-wasting illness in two young boys, and could prove effective against other genetic disorders, researchers reported.

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The STOP ALD Foundation Applauds Gene Therapy Success In Severe Brain Disorder

The Stop ALD Foundation has applauded the investigators who are reporting in the current issue of Science successful results from the pioneering use of gene therapy for adrenoleukodystrophy (ALD), a potentially crippling and fatal brain disorder in young boys.

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Gene Therapy Success for Brian Disease

Naturenews reports on another important success for gene therapy-- treatment for ALD ( adrenoleukodystrophy ), a rare, inherited metabolic disorder that afflicts young males. ALD results in severe degeneration of the structure that is crucial for brain-cell function and most die before adolescence. Here is a sample from the news story: Researchers have halted a fatal brain disease...

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New gene therapy used to treat rare brain disease

French scientists mixed gene therapy and bone-marrow transplants in two boys to seemingly halt a brain disease that can kill by adolescence.

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Nanoparticles for gene therapy improve

(PhysOrg.com) -- About five years ago, Professor Janet Sawicki at the Lankenau Institute in Pennsylvania read an article about nanoparticles developed by MIT's Robert Langer for gene therapy, the insertion of genes into living cells for the treatment of disease. Sawicki was working on treating ovarian cancer by delivering -- through viruses -- the gene for the diphtheria...

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Gene Therapy Success In Severe Brain Disorder Applauded By The STOP ALD Foundation

Affected families achieve goal of saving children.

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New gene therapy halts 2 boys' rare brain disease

WASHINGTON -- French scientists mixed gene therapy and bone marrow transplants in two boys to seemingly halt a brain disease that can kill by adolescence. The surprise ingredient: They disabled the HIV virus so it couldn't cause AIDS, and then used it to carry in the healthy new gene.

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SCIENCE: Gene Therapy Slows Brain Disease

Science : Gene Therapy Technique Slows “Lorenzo’s Oil” Brain Disease An international team of researchers combined gene therapy with blood stem cell therapy to treat X-linked ALD, a fatal brain disease featured in the movie Lorenzo’s Oil .

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Brain disease treated by gene therapy

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Gene therapy can cure brain disease

In what could raise hopes of thousands of families, scientists have managed to halt a rare brain disease that kills boys by the time of adolescence through an experimental gene therapy.