Gene therapy strengthens muscles in monkeys

2010-04-16T09:13:53Z

WASHINGTON (Reuters) - A gene therapy treatment that stops the breakdown of muscle appeared safe in monkeys and may build up muscle, too, researchers reported on Wednesday.

The approach is being developed in the hope of treating severe muscle weakness caused by multiple sclerosis, muscular dystrophy and neurodegenerative diseases, but progress is slow in part because of fears about its safety.

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Scientists halt brain disease with new gene therapy

2010-04-16T09:21:03Z

LONDON (Reuters) - Scientists have managed to halt a rare and fatal brain disease with an experimental gene therapy technique using a deactivated version of the AIDS virus, a study published on Thursday showed.

The international team used a disabled form of human immunodeficiency virus (HIV) to deliver working genes to two boys with the brain disease X-linked adrenoleukodystrophy (ALD). Their success may help shape f...

Gene therapy beats back brain wasting disease: study

2010-04-16T09:22:41Z

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 Thursday.

The boys, both seven when treatment began in France, are beset with X-linked adrenoleukodystrophy, or ALD, a hereditary brain disease that slowly strips away layers of fatty acids protecting nerve fibres in the brain.

Without treatment, ALD...

New gene therapy halts 2 boys' rare brain disease

2010-04-16T09:22:47Z

French scientists appear to halt rare brain disease in 2 boys with new gene therapy approach

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.

The experiment marks the first time researchers have tried that long-contemplated step in people ...

Gene therapy experiment restores sight in a few

2010-04-16T09:39:54Z

PHILADELPHIA (Reuters) - Nine-year-old Corey Haas can ride his bike alone now, thanks to an experimental gene therapy that has boosted his fading vision with a single treatment.

The gene therapy helped improve worsening eyesight caused by a rare inherited disease called Leber congenital amaurosis, or LCA, which makes most patients blind by age 40.

Twelve t...

Long-term monkey tests back Oxford's gene therapy

2010-04-16T09:50:28Z

LONDON (Reuters) - Long-term tests on monkeys using Oxford BioMedica's gene therapy ProSavin suggest it can treat Parkinson's disease without causing the jerky, involuntary movements associated with current drugs, researchers said on Wednesday.

Parkinson's is caused by lack of the brain chemical dopamine. Standard treatment involves oral drugs th...

Gene therapy cures form of 'bubble boy disease'

2010-04-16T17:21:12Z

Gene therapy cures children with 'bubble boy' disease; hope for other blood disorders seen

Gene therapy seems to have cured eight of 10 children who had potentially fatal "bubble boy disease," according to a study that followed their progress for about four years after treatment. The eight patients were no longer on medication for the rare disease, which cripples the body's defenses against infection. The successful treatment is reported in Thursday's issue of the...

Gene Therapy May One Day Prevent AIDS–Related Brain–Cell Death

2010-04-16T17:57:41Z

Scientists have shown that gene therapy has potential for treating brain pathology triggered by the human immunodeficiency virus (HIV), which causes AIDS. Although brain damage is among the most common complications of AIDS, there is no recognized therapy for preventing the underlying HIV-induced cell death.

NIMH-funded researchers Dr. D.S. Strayer and colleagues from National Eye Institute (NEI), part of the National Institutes of Health. In addition no adverse effects from the therapy were reported. T...

Researchers Report Progress in Salivary Gland Gene Transfer

2010-04-16T17:57:00Z

Two years ago, scientists reported exciting news in the development of gene therapy for the salivary gland. They had constructed a new version of a gene-carrying vehicle, or vector, that functioned well in the salivary glands of mice for several weeks. Most significantly, the vector - a stripped down, bioengineered version of the harmless adeno-associated virus (AAV) - had done so without triggering a sustained immune response, a common setback in gene therapy experiments.

Left unansw...

Gene Transfer Successful in Mouse Model of Limb-Girdle Muscular Dystrophy

2010-09-01T12:16:39Z

Gene Transfer Successful in Mouse Model of Limb-Girdle Muscular Dystrophy . Scientists have found that progression of limb-girdle muscular dystrophy type 2D (LGMD-2D) can be prevented over the long term in mice after transferring a correctly functioning form of the alpha-sarcoglycan gene (SGCA) to their muscle tissue. The research was partly supported by the

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