NewsGENESSaturday, March 28, 2026 · March 28, 2026
Adeno-Associated Virus Toxicity in Duchenne Muscular Dystrophy: Mechanisms and Clinical Considerations.
WHY IT MATTERS
Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
Background/Objectives : Recombinant adeno-associated virus (AAV) vectors have revolutionized gene therapy for monogenic diseases such as Duchenne muscular dystrophy (DMD). However, high systemic doses required for muscle transduction cause a spectrum of toxicities ranging from transient hepatic infl...
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