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Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
Lattanzi A, Camarena J, Lahiri P, Segal H, Srifa W, Vakulskas CA, Frock RL, Kenrick J, Lee C, Talbott N, Skowronski J, Cromer MK, Charlesworth CT, Bak RO, Mantri S, Bao G, DiGiusto D, Tisdale J, Wright JF, Bhatia N, Roncarolo MG, Dever DP, Porteus MH. Lattanzi A, et al. Among authors: mantri s. Sci Transl Med. 2021 Jun 16;13(598):eabf2444. doi: 10.1126/scitranslmed.abf2444. Sci Transl Med. 2021. PMID: 34135108 Free PMC article.
CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.
Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE, Pavel-Dinu M, Saxena N, Wilkens AB, Mantri S, Uchida N, Hendel A, Narla A, Majeti R, Weinberg KI, Porteus MH. Dever DP, et al. Among authors: mantri s. Nature. 2016 Nov 17;539(7629):384-389. doi: 10.1038/nature20134. Epub 2016 Nov 7. Nature. 2016. PMID: 27820943 Free PMC article.
Gene correction for SCID-X1 in long-term hematopoietic stem cells.
Pavel-Dinu M, Wiebking V, Dejene BT, Srifa W, Mantri S, Nicolas CE, Lee C, Bao G, Kildebeck EJ, Punjya N, Sindhu C, Inlay MA, Saxena N, DeRavin SS, Malech H, Roncarolo MG, Weinberg KI, Porteus MH. Pavel-Dinu M, et al. Among authors: mantri s. Nat Commun. 2019 Apr 9;10(1):1634. doi: 10.1038/s41467-019-09614-y. Nat Commun. 2019. PMID: 30967552 Free PMC article.
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: mantri s. Nat Commun. 2019 Sep 6;10(1):4045. doi: 10.1038/s41467-019-11962-8. Nat Commun. 2019. PMID: 31492863 Free PMC article.
Identification of preexisting adaptive immunity to Cas9 proteins in humans.
Charlesworth CT, Deshpande PS, Dever DP, Camarena J, Lemgart VT, Cromer MK, Vakulskas CA, Collingwood MA, Zhang L, Bode NM, Behlke MA, Dejene B, Cieniewicz B, Romano R, Lesch BJ, Gomez-Ospina N, Mantri S, Pavel-Dinu M, Weinberg KI, Porteus MH. Charlesworth CT, et al. Among authors: mantri s. Nat Med. 2019 Feb;25(2):249-254. doi: 10.1038/s41591-018-0326-x. Epub 2019 Jan 28. Nat Med. 2019. PMID: 30692695 Free PMC article.
Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells.
Pavel-Dinu M, Wiebking V, Dejene BT, Srifa W, Mantri S, Nicolas CE, Lee C, Bao G, Kildebeck EJ, Punjya N, Sindhu C, Inlay MA, Saxena N, DeRavin SS, Malech H, Roncarolo MG, Weinberg KI, Porteus MH. Pavel-Dinu M, et al. Among authors: mantri s. Nat Commun. 2019 Dec 4;10(1):5624. doi: 10.1038/s41467-019-13620-5. Nat Commun. 2019. PMID: 31796738 Free PMC article.
Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells.
Pavel-Dinu M, Wiebking V, Dejene BT, Srifa W, Mantri S, Nicolas CE, Lee C, Bao G, Kildebeck EJ, Punjya N, Sindhu C, Inlay MA, Saxena N, DeRavin SS, Malech H, Roncarolo MG, Weinberg KI, Porteus MH. Pavel-Dinu M, et al. Among authors: mantri s. Nat Commun. 2019 Apr 26;10(1):2021. doi: 10.1038/s41467-019-10080-9. Nat Commun. 2019. PMID: 31028274 Free PMC article.
246 results