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Clinical trial for muscular dystrophy demonstrates safety of customized gene therapy
Date:11/30/2011

ular dystrophy and is the ultimate cause of the disease's progressive muscle weakness. The replacement genes were injected into the bicep in one arm and a placebo was injected into the other arm of each of the patients. The researchers were able to detect the new genes in all of the patients treated with the gene therapy, but no immunological response.

As they move on to the next phase of clinical trials, Samulski says they are carefully considering how best to administer the gene therapy vectors to patients. Delivering enough replacement genes to a therapeutic effect could require larger doses of virus, which in turn could elicit an unwanted immune response. So the researchers are exploring a number of different options, including using a new high pressure technique developed by William J. Powers, MD, professor and chair of neurology at UNC, reported last July in the same journal, to get the virus into muscle at lower doses.


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Contact: Les Lang
llang@med.unc.edu
919-966-9366
University of North Carolina School of Medicine
Source:Eurekalert  

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Clinical trial for muscular dystrophy demonstrates safety of customized gene therapy
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