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Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency.
Fañanas-Baquero S, Quintana-Bustamante O, Dever DP, Alberquilla O, Sanchez-Dominguez R, Camarena J, Ojeda-Perez I, Dessy-Rodriguez M, Turk R, Schubert MS, Lattanzi A, Xu L, Lopez-Lorenzo JL, Bianchi P, Bueren JA, Behlke MA, Porteus M, Segovia JC. Fañanas-Baquero S, et al. Among authors: behlke ma. Mol Ther Methods Clin Dev. 2021 May 14;22:237-248. doi: 10.1016/j.omtm.2021.05.001. eCollection 2021 Sep 10. Mol Ther Methods Clin Dev. 2021. PMID: 34485608 Free PMC article.
A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells.
Vakulskas CA, Dever DP, Rettig GR, Turk R, Jacobi AM, Collingwood MA, Bode NM, McNeill MS, Yan S, Camarena J, Lee CM, Park SH, Wiebking V, Bak RO, Gomez-Ospina N, Pavel-Dinu M, Sun W, Bao G, Porteus MH, Behlke MA. Vakulskas CA, et al. Among authors: behlke ma. Nat Med. 2018 Aug;24(8):1216-1224. doi: 10.1038/s41591-018-0137-0. Epub 2018 Aug 6. Nat Med. 2018. PMID: 30082871 Free PMC article.
Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells.
Cromer MK, Camarena J, Martin RM, Lesch BJ, Vakulskas CA, Bode NM, Kurgan G, Collingwood MA, Rettig GR, Behlke MA, Lemgart VT, Zhang Y, Goyal A, Zhao F, Ponce E, Srifa W, Bak RO, Uchida N, Majeti R, Sheehan VA, Tisdale JF, Dever DP, Porteus MH. Cromer MK, et al. Among authors: behlke ma. Nat Med. 2021 Apr;27(4):677-687. doi: 10.1038/s41591-021-01284-y. Epub 2021 Mar 18. Nat Med. 2021. PMID: 33737751 Free PMC article.
CRISPAltRations: a validated cloud-based approach for interrogation of double-strand break repair mediated by CRISPR genome editing.
Kurgan G, Turk R, Li H, Roberts N, Rettig GR, Jacobi AM, Tso L, Sturgeon M, Mertens M, Noten R, Florus K, Behlke MA, Wang Y, McNeill MS. Kurgan G, et al. Among authors: behlke ma. Mol Ther Methods Clin Dev. 2021 Apr 2;21:478-491. doi: 10.1016/j.omtm.2021.03.024. eCollection 2021 Jun 11. Mol Ther Methods Clin Dev. 2021. PMID: 33981780 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: behlke ma. 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.
AsCas12a ultra nuclease facilitates the rapid generation of therapeutic cell medicines.
Zhang L, Zuris JA, Viswanathan R, Edelstein JN, Turk R, Thommandru B, Rube HT, Glenn SE, Collingwood MA, Bode NM, Beaudoin SF, Lele S, Scott SN, Wasko KM, Sexton S, Borges CM, Schubert MS, Kurgan GL, McNeill MS, Fernandez CA, Myer VE, Morgan RA, Behlke MA, Vakulskas CA. Zhang L, et al. Among authors: behlke ma. Nat Commun. 2021 Jun 23;12(1):3908. doi: 10.1038/s41467-021-24017-8. Nat Commun. 2021. PMID: 34162850 Free PMC article.
Large-scale GMP-compliant CRISPR-Cas9-mediated deletion of the glucocorticoid receptor in multivirus-specific T cells.
Basar R, Daher M, Uprety N, Gokdemir E, Alsuliman A, Ensley E, Ozcan G, Mendt M, Hernandez Sanabria M, Kerbauy LN, Nunez Cortes AK, Li L, Banerjee PP, Muniz-Feliciano L, Acharya S, Fowlkes NW, Lu J, Li S, Mielke S, Kaplan M, Nandivada V, Bdaiwi M, Kontoyiannis AD, Li Y, Liu E, Ang S, Marin D, Brunetti L, Gundry MC, Turk R, Schubert MS, Rettig GR, McNeill MS, Kurgan G, Behlke MA, Champlin R, Shpall EJ, Rezvani K. Basar R, et al. Among authors: behlke ma. Blood Adv. 2020 Jul 28;4(14):3357-3367. doi: 10.1182/bloodadvances.2020001977. Blood Adv. 2020. PMID: 32717029 Free PMC article.
150 results