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Trial Of DMD Gene Therapy Initiated At Ohio

as miniatured while retaining at the same time the crucial elements of the DNA.

AskBio’s Biostrophin therapy delivers the miniaturized replacement dystrophin gene with the help of Biological Nano Particles (BNP). BNP’s carry the minidystrophin gene to muscle cells. The Food and Drug Administration gave the team a green light to proceed with the human trial on March 3. The trial will test six boys with DMD, each of whom will receive minidystrophin genes in a muscle of one arm and a placebo in the other arm. Neither the investigators nor the participants will know which muscle got the dystrophin genes. After several weeks, an analysis of the injected muscle tissue’s microscopic appearance, as well as extensive testing of the health and strength of the trial participants would confirm the effectiveness of the therapy.
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