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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: morris ca. Sci Transl Med. 2023 Jan 4;15(677):eabo1815. doi: 10.1126/scitranslmed.abo1815. Epub 2023 Jan 4. Sci Transl Med. 2023. PMID: 36599002 Free PMC article.
Dystrophin-deficient dogs with reduced myostatin have unequal muscle growth and greater joint contractures.
Kornegay JN, Bogan DJ, Bogan JR, Dow JL, Wang J, Fan Z, Liu N, Warsing LC, Grange RW, Ahn M, Balog-Alvarez CJ, Cotten SW, Willis MS, Brinkmeyer-Langford C, Zhu H, Palandra J, Morris CA, Styner MA, Wagner KR. Kornegay JN, et al. Among authors: morris ca. Skelet Muscle. 2016 Apr 4;6:14. doi: 10.1186/s13395-016-0085-7. eCollection 2016. Skelet Muscle. 2016. PMID: 27047655 Free PMC article.
Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients.
Paquin RS, Fischer R, Mansfield C, Mange B, Beaverson K, Ganot A, Martin AS, Morris C, Rensch C, Ricotti V, Russo LJ, Sadosky A, Smith EC, Peay HL. Paquin RS, et al. Orphanet J Rare Dis. 2019 May 9;14(1):102. doi: 10.1186/s13023-019-1069-6. Orphanet J Rare Dis. 2019. PMID: 31072340 Free PMC article.
Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice.
Hamm SE, Fathalikhani DD, Bukovec KE, Addington AK, Zhang H, Perry JB, McMillan RP, Lawlor MW, Prom MJ, Vanden Avond MA, Kumar SN, Coleman KE, Dupont JB, Mack DL, Brown DA, Morris CA, Gonzalez JP, Grange RW. Hamm SE, et al. Among authors: morris ca. Mol Ther Methods Clin Dev. 2021 Mar 3;21:144-160. doi: 10.1016/j.omtm.2021.02.024. eCollection 2021 Jun 11. Mol Ther Methods Clin Dev. 2021. PMID: 33850950 Free PMC article.
Anti-latent TGFβ binding protein 4 antibody improves muscle function and reduces muscle fibrosis in muscular dystrophy.
Demonbreun AR, Fallon KS, Oosterbaan CC, Vaught LA, Reiser NL, Bogdanovic E, Velez MP, Salamone IM, Page PGT, Hadhazy M, Quattrocelli M, Barefield DY, Wood LD, Gonzalez JP, Morris C, McNally EM. Demonbreun AR, et al. Sci Transl Med. 2021 Sep 8;13(610):eabf0376. doi: 10.1126/scitranslmed.abf0376. Epub 2021 Sep 8. Sci Transl Med. 2021. PMID: 34516828 Free PMC article.
360 results