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Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I.
Gomez-Ospina N, Scharenberg SG, Mostrel N, Bak RO, Mantri S, Quadros RM, Gurumurthy CB, Lee C, Bao G, Suarez CJ, Khan S, Sawamoto K, Tomatsu S, Raj N, Attardi LD, Aurelian L, Porteus MH. Gomez-Ospina N, et al. Among authors: scharenberg sg. Nat Commun. 2019 Sep 6;10(1):4045. doi: 10.1038/s41467-019-11962-8. Nat Commun. 2019. PMID: 31492863 Free PMC article.
CRISPR/Cas9 Genome Engineering in Engraftable Human Brain-Derived Neural Stem Cells.
Dever DP, Scharenberg SG, Camarena J, Kildebeck EJ, Clark JT, Martin RM, Bak RO, Tang Y, Dohse M, Birgmeier JA, Jagadeesh KA, Bejerano G, Tsukamoto A, Gomez-Ospina N, Uchida N, Porteus MH. Dever DP, et al. Among authors: scharenberg sg. iScience. 2019 May 31;15:524-535. doi: 10.1016/j.isci.2019.04.036. Epub 2019 May 4. iScience. 2019. PMID: 31132746 Free PMC article.