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AAV-based in vivo gene therapy for neurological disorders.
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.
Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease.
Herstine JA, Chang PK, Chornyy S, Stevenson TJ, Sunshine AC, Nokhrina K, Rediger J, Wentz J, Vetter TA, Scholl E, Holaway C, Pyne NK, Bratasz A, Yeoh S, Flanigan KM, Bonkowsky JL, Bradbury AM.
Herstine JA, et al.
Mol Ther. 2024 Mar 27:S1525-0016(24)00212-0. doi: 10.1016/j.ymthe.2024.03.034. Online ahead of print.
Mol Ther. 2024.
PMID: 38549375
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