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Comment
. 2024 Jan 2;134(1):e177078.
doi: 10.1172/JCI177078.

Improving the safety of systemic viral gene therapy

Elizabeth M McNally
Comment

Improving the safety of systemic viral gene therapy

Elizabeth M McNally. J Clin Invest. .
No abstract available

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Conflict of interest statement

Conflict of interest: EMM has been or is a consultant to Amgen, AstraZeneca, Avidity Biosciences, Cytokinetics, 4D Molecular Therapeutics, Pfizer, and Tenaya Therapeutics. EMM is also a founder of Ikaika Therapeutics.

Comment on

References

    1. FDA. Approved Cellular and Gene Therapy Producsts. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-produ... Updated June 30, 2023. Accessed November 6, 2023.
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    1. Lek A, et al. Meeting report: 2022 Muscular Dystrophy Association summit on ‘safety and challenges in gene transfer therapy’. J Neuromuscul Dis. 2023;10(3):327–336. doi: 10.3233/JND-221639. - DOI - PubMed
    1. Lek A, et al. Death after high-dose rAAV9 gene therapy in a patient with Duchenne’s muscular dystrophy. N Engl J Med. 2023;389(13):1203–1210. doi: 10.1056/NEJMoa2307798. - DOI - PMC - PubMed
    1. Bönnemann CG, et al. Dystrophin immunity after gene therapy for Duchenne’s muscular dystrophy. N Engl J Med. 2023;388(24):2294–2296. doi: 10.1056/NEJMc2212912. - DOI - PubMed

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