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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: pyle ad. 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.
Exon Skipping Therapy.
Young CS, Pyle AD. Young CS, et al. Among authors: pyle ad. Cell. 2016 Nov 17;167(5):1144. doi: 10.1016/j.cell.2016.10.050. Cell. 2016. PMID: 27863231 Free article.
CRISPR for Neuromuscular Disorders: Gene Editing and Beyond.
Young CS, Pyle AD, Spencer MJ. Young CS, et al. Among authors: pyle ad. Physiology (Bethesda). 2019 Sep 1;34(5):341-353. doi: 10.1152/physiol.00012.2019. Physiology (Bethesda). 2019. PMID: 31389773 Free PMC article. Review.
A Human Skeletal Muscle Atlas Identifies the Trajectories of Stem and Progenitor Cells across Development and from Human Pluripotent Stem Cells.
Xi H, Langerman J, Sabri S, Chien P, Young CS, Younesi S, Hicks M, Gonzalez K, Fujiwara W, Marzi J, Liebscher S, Spencer M, Van Handel B, Evseenko D, Schenke-Layland K, Plath K, Pyle AD. Xi H, et al. Among authors: pyle ad. Cell Stem Cell. 2020 Jul 2;27(1):158-176.e10. doi: 10.1016/j.stem.2020.04.017. Epub 2020 May 11. Cell Stem Cell. 2020. PMID: 32396864 Free PMC article.
64 results