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Intrathecal Gene Therapy for Giant Axonal Neuropathy.
Bharucha-Goebel DX, Todd JJ, Saade D, Norato G, Jain M, Lehky T, Bailey RM, Chichester JA, Calcedo R, Armao D, Foley AR, Mohassel P, Tesfaye E, Carlin BP, Seremula B, Waite M, Zein WM, Huryn LA, Crawford TO, Sumner CJ, Hoke A, Heiss JD, Charnas L, Hooper JE, Bouldin TW, Kang EM, Rybin D, Gray SJ, Bönnemann CG; GAN Trial Team. Bharucha-Goebel DX, et al. Among authors: gray sj. N Engl J Med. 2024 Mar 21;390(12):1092-1104. doi: 10.1056/NEJMoa2307952. N Engl J Med. 2024. PMID: 38507752
AAV-based in vivo gene therapy for neurological disorders.
Ling Q, Herstine JA, Bradbury A, Gray SJ. Ling Q, et al. Among authors: gray sj. Nat Rev Drug Discov. 2023 Oct;22(10):789-806. doi: 10.1038/s41573-023-00766-7. Epub 2023 Sep 1. Nat Rev Drug Discov. 2023. PMID: 37658167 Review.
308 results