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Article

Promoting expression in gene therapy: more is not always better

27 Feb 2024

Abstract excerpt

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by loss-of-function of SMN1. SMA is characterized by degeneration of motor neurons in the spinal cord, leading to progressive muscle weakness and atrophy. One of three currently available treatments is onasemnogene abeparvovec, an AAV9-based gene replacement therapy. Despite its effectiveness in improving motor function in SMA...

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PMID: 38413837
DOI: 10.1038/s44321-024-00036-y

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