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Emerging therapies in hereditary ataxias.
Eisel MLS, Burns M, Ashizawa T, Byrne B, Corti M, Subramony SH. Eisel MLS, et al. Among authors: byrne b. Trends Mol Med. 2024 Aug 16:S1471-4914(24)00194-1. doi: 10.1016/j.molmed.2024.07.008. Online ahead of print. Trends Mol Med. 2024. PMID: 39153956 Review.
Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation.
Corti M, Byrne BJ, Gessler DJ, Thompson G, Norman S, Lammers J, Coleman KE, Liberati C, Elder ME, Escolar ML, Tuna IS, Mesaros C, Kleiner GI, Barbouth DS, Gray-Edwards HL, Clement N, Cleaver BD, Gao G. Corti M, et al. Mol Ther Methods Clin Dev. 2023 Jun 19;30:303-314. doi: 10.1016/j.omtm.2023.06.001. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37601414 Free PMC article.
Current avenues of gene therapy in Pompe disease.
Leon-Astudillo C, Trivedi PD, Sun RC, Gentry MS, Fuller DD, Byrne BJ, Corti M. Leon-Astudillo C, et al. Curr Opin Neurol. 2023 Oct 1;36(5):464-473. doi: 10.1097/WCO.0000000000001187. Epub 2023 Jul 19. Curr Opin Neurol. 2023. PMID: 37639402 Free PMC article. Review.
Perspectives of the Friedreich ataxia community on gene therapy clinical trials.
Trantham SJ, Coker MA, Norman S, Crowley E, Berthy J, Byrne BJ, Subramony S, Lou X, Corti M. Trantham SJ, et al. Mol Ther Methods Clin Dev. 2023 Dec 18;32(1):101179. doi: 10.1016/j.omtm.2023.101179. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2023. PMID: 38261944 Free PMC article.
Neurological glycogen storage diseases and emerging therapeutics.
Colpaert M, Singh PK, Donohue KJ, Pires NT, Fuller DD, Corti M, Byrne BJ, Sun RC, Vander Kooi CW, Gentry MS. Colpaert M, et al. Neurotherapeutics. 2024 Sep;21(5):e00446. doi: 10.1016/j.neurot.2024.e00446. Epub 2024 Sep 14. Neurotherapeutics. 2024. PMID: 39277505 Free PMC article. Review.
991 results