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Year Number of Results
2009 1
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2013 2
2014 1
2015 5
2016 5
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2020 4
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2023 7
2024 4

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Page 1
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. Among authors: corti m. 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.
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. Among authors: corti m. 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 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.
The multifaceted roles of the brain glycogen.
Markussen KH, Corti M, Byrne BJ, Vander Kooi CW, Sun RC, Gentry MS. Markussen KH, et al. Among authors: corti m. J Neurochem. 2023 Aug 9:10.1111/jnc.15926. doi: 10.1111/jnc.15926. Online ahead of print. J Neurochem. 2023. PMID: 37554056 Review.
Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.
Birch SM, Lawlor MW, Conlon TJ, Guo LJ, Crudele JM, Hawkins EC, Nghiem PP, Ahn M, Meng H, Beatka MJ, Fickau BA, Prieto JC, Styner MA, Struharik MJ, Shanks C, Brown KJ, Golebiowski D, Bettis AK, Balog-Alvarez CJ, Clement N, Coleman KE, Corti M, Pan X, Hauschka SD, Gonzalez JP, Morris CA, Schneider JS, Duan D, Chamberlain JS, Byrne BJ, Kornegay JN. Birch SM, et al. Among authors: corti m. Sci Transl Med. 2023 Jan 4;15(677):eabo1815. doi: 10.1126/scitranslmed.abo1815. Epub 2023 Jan 4. Sci Transl Med. 2023. PMID: 36599002
39 results