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Experimental gene therapy treatment for Duchenne muscular dystrophy offers hope for youngster
Date:2/7/2013

hin in the participants' muscles the latter results obtained through muscle biopsies at several times during study. Of particular interest to the research teams are the residual effects of the drug several weeks after the injection series is completed.

Although McDonald says it is too early to draw conclusions from the current clinical trial, he suggests there is reason to be optimistic based on animal studies and evidence from another clinical trial in Europe. In that trial, patients and study investigators knew which patients were actually taking the experimental drug, meaning it was an "open" study. The current U.S. study is more rigorous because doctors and families are "blinded" about which drug regimen each participant has -- either one of two dosages of the drug or a placebo -- until the end of the study.

According to McDonald, the antisense oligonucleotide-mediated genetic therapy approach is particularly exciting because of its potential applications to most other variants of Duchenne muscular dystrophy and other genetic diseases. Although the current experimental therapy specifically addresses mutations in only one particular region of the gene, the same therapeutic concept can be applied to many others.

"If successful, this approach can be developed into specific gene therapy that represents truly personalized medicine," said McDonald. "Covering a mutation to restore a normal genetic open reading frame for protein synthesis can be a powerful approach for a variety of genetic diseases."

Standardizing the drug as a therapy for Duchenne patients still requires approval by the U.S. Food and Drug Administration. Investigators say the therapy could be similar to insulin injections for diabetes. But rather than daily injections, the Duchenne treatment might only involve weekly shots, which youngsters could learn to administer on their own.

Now that the clinical trial is closed at UC Davis to new enrollees, McDonald
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Contact: Charles Casey
charles.casey@ucdmc.ucdavis.edu
916-734-9048
University of California - Davis Health System
Source:Eurekalert

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