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Emerging genetic therapies to treat Duchenne muscular dystrophy.
Nelson SF, Crosbie RH, Miceli MC, Spencer MJ. Nelson SF, et al. Among authors: miceli mc. Curr Opin Neurol. 2009 Oct;22(5):532-8. doi: 10.1097/WCO.0b013e32832fd487. Curr Opin Neurol. 2009. PMID: 19745732 Free PMC article. Review.
A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells.
Young CS, Hicks MR, Ermolova NV, Nakano H, Jan M, Younesi S, Karumbayaram S, Kumagai-Cresse C, Wang D, Zack JA, Kohn DB, Nakano A, Nelson SF, Miceli MC, Spencer MJ, Pyle AD. Young CS, et al. Among authors: miceli mc. Cell Stem Cell. 2016 Apr 7;18(4):533-40. doi: 10.1016/j.stem.2016.01.021. Epub 2016 Feb 11. Cell Stem Cell. 2016. PMID: 26877224 Free PMC article.
FDA Approval of Eteplirsen for Muscular Dystrophy.
Nelson SF, Miceli MC. Nelson SF, et al. Among authors: miceli mc. JAMA. 2017 Apr 11;317(14):1480. doi: 10.1001/jama.2017.2601. JAMA. 2017. PMID: 28399245 No abstract available.
DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype.
Wang RT, Barthelemy F, Martin AS, Douine ED, Eskin A, Lucas A, Lavigne J, Peay H, Khanlou N, Sweeney L, Cantor RM, Miceli MC, Nelson SF. Wang RT, et al. Among authors: miceli mc. Hum Mutat. 2018 Sep;39(9):1193-1202. doi: 10.1002/humu.23561. Epub 2018 Jul 12. Hum Mutat. 2018. PMID: 29907980 Free PMC article.
64 results