Gene replacement therapies have shown remarkable advances recently, including onasemnogene abeparvovec for treatment of symptomatic and presymptomatic patients with spinal muscular atrophy. A recent report by Van Alstyne and colleagues in a mouse model of spinal muscular atrophy raises concerns that such treatment can generate toxic overexpression of SMN protein.
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R.S.F. is an advisor to and investigator in clinical trials sponsored by AveXis/Novartis Gene Therapies, Biogen, and Roche; and has been an advisor to Scholar Rock, Cure SMA, SMA Europe, and the n-Lorem Foundation. K.H.F. has been an unpaid consultant and collaborator with Ionis, Biogen, Novartis, and the SMA Foundation.
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Finkel, R.S., Fischbeck, K.H. Maybe too much of a good thing in gene therapy. Nat Neurosci 24, 901–902 (2021). https://doi.org/10.1038/s41593-021-00882-w
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DOI: https://doi.org/10.1038/s41593-021-00882-w
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