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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. 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. Cell. 2016 Nov 17;167(5):1144. doi: 10.1016/j.cell.2016.10.050. Cell. 2016. PMID: 27863231 Free article.
"Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic.
Gordish-Dressman H, Willmann R, Dalle Pazze L, Kreibich A, van Putten M, Heydemann A, Bogdanik L, Lutz C, Davies K, Demonbreun AR, Duan D, Elsey D, Fukada SI, Girgenrath M, Patrick Gonzalez J, Grounds MD, Nichols A, Partridge T, Passini M, Sanarica F, Schnell FJ, Wells DJ, Yokota T, Young CS, Zhong Z, Spurney C, Spencer M, De Luca A, Nagaraju K, Aartsma-Rus A. Gordish-Dressman H, et al. Among authors: young cs. J Neuromuscul Dis. 2018;5(4):407-417. doi: 10.3233/JND-180324. J Neuromuscul Dis. 2018. PMID: 30198876 Free PMC article.
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: young cs. 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.
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: young cs. Cell Stem Cell. 2020 Jul 2;27(1):181-185. doi: 10.1016/j.stem.2020.06.006. Cell Stem Cell. 2020. PMID: 32619514 Free PMC article. No abstract available.
154 results