Gene therapy for primary mitochondrial diseases: experimental advances and clinical challenges
Author:
Publisher
Springer Science and Business Media LLC
Subject
Cellular and Molecular Neuroscience,Neurology (clinical)
Link
https://www.nature.com/articles/s41582-022-00715-9.pdf
Reference114 articles.
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3. Thompson, A. A. et al. Gene therapy in patients with transfusion-dependent β-thalassemia. N. Engl. J. Med. 378, 1479–1493 (2018).
4. Kuzmin, D. A. et al. The clinical landscape for AAV gene therapies. Nat. Rev. Drug. Discov. 20, 173–174 (2021).
5. Iftikhar, M., Frey, J., Shohan, M. J., Malek, S. & Mousa, S. A. Current and emerging therapies for Duchenne muscular dystrophy and spinal muscular atrophy. Pharmacol. Ther. 220, 107719 (2021).
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