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Year Number of Results
2009 1
2012 2
2013 2
2014 1
2015 5
2016 5
2017 2
2018 1
2019 2
2020 4
2021 7
2022 4
2023 7
2024 4

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Page 1
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Among authors: corti m. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.
Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study).
Lynch DR, Chin MP, Delatycki MB, Subramony SH, Corti M, Hoyle JC, Boesch S, Nachbauer W, Mariotti C, Mathews KD, Giunti P, Wilmot G, Zesiewicz T, Perlman S, Goldsberry A, O'Grady M, Meyer CJ. Lynch DR, et al. Among authors: corti m. Ann Neurol. 2021 Feb;89(2):212-225. doi: 10.1002/ana.25934. Epub 2020 Nov 5. Ann Neurol. 2021. PMID: 33068037 Free PMC article. Clinical Trial.
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
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.
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.
Considerations for Systemic Use of Gene Therapy.
Byrne BJ, Corti M, Muntoni F. Byrne BJ, et al. Among authors: corti m. Mol Ther. 2021 Feb 3;29(2):422-423. doi: 10.1016/j.ymthe.2021.01.016. Epub 2021 Jan 14. Mol Ther. 2021. PMID: 33485465 Free PMC article. Review. No abstract available.
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.
Clinical management guidelines for Friedreich ataxia: best practice in rare diseases.
Corben LA, Collins V, Milne S, Farmer J, Musheno A, Lynch D, Subramony S, Pandolfo M, Schulz JB, Lin K, Delatycki MB; Clinical Management Guidelines Writing Group. Corben LA, et al. Orphanet J Rare Dis. 2022 Nov 12;17(1):415. doi: 10.1186/s13023-022-02568-3. Orphanet J Rare Dis. 2022. PMID: 36371255 Free PMC article.
39 results