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14 results

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Page 1
Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency.
Pavel-Dinu M, Gardner CL, Nakauchi Y, Kawai T, Delmonte OM, Palterer B, Bosticardo M, Pala F, Viel S, Malech HL, Ghanim HY, Bode NM, Kurgan GL, Detweiler AM, Vakulskas CA, Neff NF, Sheikali A, Menezes ST, Chrobok J, Hernández González EM, Majeti R, Notarangelo LD, Porteus MH. Pavel-Dinu M, et al. Among authors: kurgan gl. Blood Adv. 2024 Apr 9;8(7):1820-1833. doi: 10.1182/bloodadvances.2023011766. Blood Adv. 2024. PMID: 38096800 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. 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.
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: kurgan gl. Nat Commun. 2021 Jun 23;12(1):3908. doi: 10.1038/s41467-021-24017-8. Nat Commun. 2021. PMID: 34162850 Free PMC article.
Author Correction: 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: kurgan gl. Nat Commun. 2021 Jul 19;12(1):4500. doi: 10.1038/s41467-021-24770-w. Nat Commun. 2021. PMID: 34282150 Free PMC article. No abstract available.
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. 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. Blood. 2021 Feb 4;137(5):624-636. doi: 10.1182/blood.2020007748. Blood. 2021. PMID: 32902645 Free PMC article.
Homology-independent targeted insertion (HITI) enables guided CAR knock-in and efficient clinical scale CAR-T cell manufacturing.
Balke-Want H, Keerthi V, Gkitsas N, Mancini AG, Kurgan GL, Fowler C, Xu P, Liu X, Asano K, Patel S, Fisher CJ, Brown AK, Tunuguntla RH, Patel S, Sotillo E, Mackall CL, Feldman SA. Balke-Want H, et al. Among authors: kurgan gl. Mol Cancer. 2023 Jun 26;22(1):100. doi: 10.1186/s12943-023-01799-7. Mol Cancer. 2023. PMID: 37365642 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. 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.
14 results