Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy Patients
Abstract
Whole-body systemic gene therapy is likely the most effective way to reduce greatly the disease burden of Duchenne muscular dystrophy (DMD), an X-linked inherited muscle disease that leads to premature death in early adulthood. Genetically, DMD is due to null mutation of the dystrophin gene, one of the largest genes in the genome. Recent studies have shown highly promising improvements in animal models with intravascular delivery of the...
Paper Details
Title
Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy Patients
Published Date
Jul 1, 2018
Journal
Volume
29
Issue
7
Pages
733 - 736
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