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Characterization of behavioral and neuromuscular junction phenotypes in a novel allelic series of SMA mouse models.
Osborne M, Gomez D, Feng Z, McEwen C, Beltran J, Cirillo K, El-Khodor B, Lin MY, Li Y, Knowlton WM, McKemy DD, Bogdanik L, Butts-Dehm K, Martens K, Davis C, Doty R, Wardwell K, Ghavami A, Kobayashi D, Ko CP, Ramboz S, Lutz C. Osborne M, et al. Hum Mol Genet. 2012 Oct 15;21(20):4431-47. doi: 10.1093/hmg/dds285. Epub 2012 Jul 16. Hum Mol Genet. 2012. PMID: 22802075 Free PMC article.
Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA.
Kong L, Valdivia DO, Simon CM, Hassinan CW, Delestrée N, Ramos DM, Park JH, Pilato CM, Xu X, Crowder M, Grzyb CC, King ZA, Petrillo M, Swoboda KJ, Davis C, Lutz CM, Stephan AH, Zhao X, Weetall M, Naryshkin NA, Crawford TO, Mentis GZ, Sumner CJ. Kong L, et al. Among authors: davis c. Sci Transl Med. 2021 Jan 27;13(578):eabb6871. doi: 10.1126/scitranslmed.abb6871. Sci Transl Med. 2021. PMID: 33504650 Free PMC article.
AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich's ataxia.
Chang JC, Ryan MR, Stark MC, Liu S, Purushothaman P, Bolan F, Johnson CA, Champe M, Meng H, Lawlor MW, Halawani S, Ngaba LV, Lynch DR, Davis C, Gonzalo-Gil E, Lutz C, Urbinati F, Medicherla B, Fonck C. Chang JC, et al. Mol Ther Methods Clin Dev. 2024 Jan 22;32(1):101193. doi: 10.1016/j.omtm.2024.101193. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38352270 Free PMC article.
263 results