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Safer Gene Therapy? Hope For Parkinson's, Alzheimer's, and MS

Date:
October 27, 2007
Source:
Cedars-Sinai Medical Center
Summary:
It is possible to sustain therapeutic gene expression in the central nervous system for up to a year, even in the presence of an anti-viral immune response mechanism that is normally present in humans, according to new research. This finding may pave the way to more effective, long lasting, and safer gene therapy treatments.
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Researchers at the Board of Governors Gene Therapeutics Research Institute at Cedars-Sinai Medical Center have shown for the first time that it is possible to sustain therapeutic gene expression in the central nervous system for up to a year, even in the presence of an anti-viral immune response mechanism that is normally present in humans.

The researchers demonstrated in an animal model that the delivery system for the gene, a novel gutted adenoviral vector called HC-Adv, is completely invisible to the immune system.

Vectors previously used to deliver genes carried minute amounts of viral proteins that were detected by the immune system, triggering an immune response that rendered the therapeutic gene inactive after a period of weeks.

According to the researchers, this delivery system is safer and more effective than what is currently available, and should therefore advance clinical gene therapy trials for people suffering from central nervous system disorders such as Parkinson's, Alzheimer's, and Multiple Sclerosis.

*Study appeared online in Molecular Therapy, the official journal of The American Society of Gene Therapy

The research was sponsored in part by The National Institutes of Health.


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The above post is reprinted from materials provided by Cedars-Sinai Medical Center. Note: Materials may be edited for content and length.


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Cedars-Sinai Medical Center. "Safer Gene Therapy? Hope For Parkinson's, Alzheimer's, and MS." ScienceDaily. ScienceDaily, 27 October 2007. <www.sciencedaily.com/releases/2007/10/071026095210.htm>.
Cedars-Sinai Medical Center. (2007, October 27). Safer Gene Therapy? Hope For Parkinson's, Alzheimer's, and MS. ScienceDaily. Retrieved July 5, 2015 from www.sciencedaily.com/releases/2007/10/071026095210.htm
Cedars-Sinai Medical Center. "Safer Gene Therapy? Hope For Parkinson's, Alzheimer's, and MS." ScienceDaily. www.sciencedaily.com/releases/2007/10/071026095210.htm (accessed July 5, 2015).

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