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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. Among authors: bradbury am. Mol Ther. 2024 Jun 5;32(6):1701-1720. doi: 10.1016/j.ymthe.2024.03.034. Epub 2024 Mar 27. Mol Ther. 2024. PMID: 38549375
Genetic separation of Brca1 functions reveal mutation-dependent Polθ vulnerabilities.
Krais JJ, Glass DJ, Chudoba I, Wang Y, Feng W, Simpson D, Patel P, Liu Z, Neumann-Domer R, Betsch RG, Bernhardy AJ, Bradbury AM, Conger J, Yueh WT, Nacson J, Pomerantz RT, Gupta GP, Testa JR, Johnson N. Krais JJ, et al. Among authors: bradbury am. Nat Commun. 2023 Nov 24;14(1):7714. doi: 10.1038/s41467-023-43446-1. Nat Commun. 2023. PMID: 38001070 Free PMC article.
Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy.
Bradbury AM, Bagel J, Swain G, Miyadera K, Pesayco JP, Assenmacher CA, Brisson B, Hendricks I, Wang XH, Herbst Z, Pyne N, Odonnell P, Shelton GD, Gelb M, Hackett N, Szabolcs P, Vite CH, Escolar M. Bradbury AM, et al. Mol Ther. 2024 Jan 3;32(1):44-58. doi: 10.1016/j.ymthe.2023.11.014. Epub 2023 Nov 11. Mol Ther. 2024. PMID: 37952085
Life-Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy.
Johnson AK, McCurdy VJ, Gray-Edwards HL, Maguire AS, Cochran JN, Gross AL, Skinner HE, Randle AN, Shirley JL, Brunson BL, Bradbury AM, Leroy SG, Hwang M, Rockwell HE, Cox NR, Baker HJ, Seyfried TN, Sena-Esteves M, Martin DR. Johnson AK, et al. Among authors: bradbury am. Ann Neurol. 2023 Nov;94(5):969-986. doi: 10.1002/ana.26756. Epub 2023 Aug 16. Ann Neurol. 2023. PMID: 37526361 Free PMC article.
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.
Davidson BL, Gao G, Berry-Kravis E, Bradbury AM, Bönnemann C, Buxbaum JD, Corcoran GR, Gray SJ, Gray-Edwards H, Kleiman RJ, Shaywitz AJ, Wang D, Zoghbi HY, Flotte TR, Tauscher-Wisniewski S, Tifft CJ, Sahin M; Gene Therapy Workshop Faculty. Davidson BL, et al. Among authors: bradbury am. Mol Ther. 2022 Jul 6;30(7):2416-2428. doi: 10.1016/j.ymthe.2022.05.014. Epub 2022 May 17. Mol Ther. 2022. PMID: 35585789 Free PMC article. Review.
Efficacy and Safety of a Krabbe Disease Gene Therapy.
Hordeaux J, Jeffrey BA, Jian J, Choudhury GR, Michalson K, Mitchell TW, Buza EL, Chichester J, Dyer C, Bagel J, Vite CH, Bradbury AM, Wilson JM. Hordeaux J, et al. Among authors: bradbury am. Hum Gene Ther. 2022 May;33(9-10):499-517. doi: 10.1089/hum.2021.245. Epub 2022 Mar 22. Hum Gene Ther. 2022. PMID: 35333110 Free PMC article.
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