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Treating neuromuscular diseases: unveiling gene therapy breakthroughs and pioneering future applications

21 Feb 2025

Abstract excerpt

In this review, we highlight recent advancements in adeno-associated virus (AAV)-based gene therapy for genetic neuromuscular diseases (NMDs), focusing on spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). We discuss the current FDA-approved gene therapies for NMDs and provide updates on preclinical studies that demonstrate the potential of various AAV-based gene therapies to reduce SMA severity...

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PMID: 39985020
DOI: 10.1186/s12929-025-01123-z

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