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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: rettig gr. 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.
Progress toward in vivo use of siRNAs-II.
Rettig GR, Behlke MA. Rettig GR, et al. Mol Ther. 2012 Mar;20(3):483-512. doi: 10.1038/mt.2011.263. Epub 2011 Dec 20. Mol Ther. 2012. PMID: 22186795 Free PMC article. Review.
Knockdown of β-catenin with dicer-substrate siRNAs reduces liver tumor burden in vivo.
Dudek H, Wong DH, Arvan R, Shah A, Wortham K, Ying B, Diwanji R, Zhou W, Holmes B, Yang H, Cyr WA, Zhou Y, Shah A, Farkiwala R, Lee M, Li Y, Rettig GR, Collingwood MA, Basu SK, Behlke MA, Brown BD. Dudek H, et al. Among authors: rettig gr. Mol Ther. 2014 Jan;22(1):92-101. doi: 10.1038/mt.2013.233. Epub 2013 Oct 3. Mol Ther. 2014. PMID: 24089139 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: rettig gr. Blood Adv. 2020 Jul 28;4(14):3357-3367. doi: 10.1182/bloodadvances.2020001977. Blood Adv. 2020. PMID: 32717029 Free PMC article.
Targeting a cytokine checkpoint enhances the fitness of armored cord blood CAR-NK cells.
Daher M, Basar R, Gokdemir E, Baran N, Uprety N, Nunez Cortes AK, Mendt M, Kerbauy LN, Banerjee PP, Shanley M, Imahashi N, Li L, Lim FLWI, Fathi M, Rezvan A, Mohanty V, Shen Y, Shaim H, Lu J, Ozcan G, Ensley E, Kaplan M, Nandivada V, Bdiwi M, Acharya S, Xi Y, Wan X, Mak D, Liu E, Jiang XR, Ang S, Muniz-Feliciano L, Li Y, Wang J, Kordasti S, Petrov N, Varadarajan N, Marin D, Brunetti L, Skinner RJ, Lyu S, Silva L, Turk R, Schubert MS, Rettig GR, McNeill MS, Kurgan G, Behlke MA, Li H, Fowlkes NW, Chen K, Konopleva M, Champlin RE, Shpall EJ, Rezvani K. Daher M, et al. Among authors: rettig gr. Blood. 2021 Feb 4;137(5):624-636. doi: 10.1182/blood.2020007748. Blood. 2021. PMID: 32902645 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: rettig gr. 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: rettig gr. 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.
20 results