An exploration of unexpected immunoreactivity to a gene-therapy product has potential implications for both understanding the disease and future gene-therapy trials.
In this study, researchers reported the results of a gene-therapy procedure tested in six children with Duchenne muscular dystrophy. Treatment involved unilateral injection of a functional miniaturized dystrophin transgene packaged within a recombinant adeno-associated virus into one biceps muscle in each patient. The study was partially manufacturer supported, and two authors are manufacturer employees.
Mini-dystrophin transgene expression in the injected muscle was transient. Hypothesizing that the cause was immune-mediated elimination of the transgene by T cells spontaneously primed to recognize dystrophin antigens, the researchers tested the patients' peripheral blood mononuclear cells for immunoreactivity to the mini-dystrophin transgen…
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DisclosuresGrant / Research supportNIH NeuroBioBank; ALS Association; NIH/National Institute of Neurological Disorders and Stroke; NIH/National Center for Advancing Translational Sciences; FDA; Department of Defense
Editorial boardsCochrane Collaboration
Leadership positions in professional societiesMuscle Study Group Executive Committee
DisclosuresGrant / Research supportNIH NeuroBioBank; ALS Association; NIH/National Institute of Neurological Disorders and Stroke; NIH/National Center for Advancing Translational Sciences; FDA; Department of Defense
Editorial boardsCochrane Collaboration
Leadership positions in professional societiesMuscle Study Group Executive Committee