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2020 1
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Page 1
Defining genome-wide CRISPR-Cas genome-editing nuclease activity with GUIDE-seq.
Malinin NL, Lee G, Lazzarotto CR, Li Y, Zheng Z, Nguyen NT, Liebers M, Topkar VV, Iafrate AJ, Le LP, Aryee MJ, Joung JK, Tsai SQ. Malinin NL, et al. Nat Protoc. 2021 Dec;16(12):5592-5615. doi: 10.1038/s41596-021-00626-x. Epub 2021 Nov 12. Nat Protoc. 2021. PMID: 34773119 Free PMC article. Review.
Deleting DNMT3A in CAR T cells prevents exhaustion and enhances antitumor activity.
Prinzing B, Zebley CC, Petersen CT, Fan Y, Anido AA, Yi Z, Nguyen P, Houke H, Bell M, Haydar D, Brown C, Boi SK, Alli S, Crawford JC, Riberdy JM, Park JJ, Zhou S, Velasquez MP, DeRenzo C, Lazzarotto CR, Tsai SQ, Vogel P, Pruett-Miller SM, Langfitt DM, Gottschalk S, Youngblood B, Krenciute G. Prinzing B, et al. Sci Transl Med. 2021 Nov 17;13(620):eabh0272. doi: 10.1126/scitranslmed.abh0272. Epub 2021 Nov 17. Sci Transl Med. 2021. PMID: 34788079 Free PMC article.
In vivo engineered B cells secrete high titers of broadly neutralizing anti-HIV antibodies in mice.
Nahmad AD, Lazzarotto CR, Zelikson N, Kustin T, Tenuta M, Huang D, Reuveni I, Nataf D, Raviv Y, Horovitz-Fried M, Dotan I, Carmi Y, Rosin-Arbesfeld R, Nemazee D, Voss JE, Stern A, Tsai SQ, Barzel A. Nahmad AD, et al. Nat Biotechnol. 2022 Aug;40(8):1241-1249. doi: 10.1038/s41587-022-01328-9. Epub 2022 Jun 9. Nat Biotechnol. 2022. PMID: 35681059 Free PMC article.
Potent and uniform fetal hemoglobin induction via base editing.
Mayuranathan T, Newby GA, Feng R, Yao Y, Mayberry KD, Lazzarotto CR, Li Y, Levine RM, Nimmagadda N, Dempsey E, Kang G, Porter SN, Doerfler PA, Zhang J, Jang Y, Chen J, Bell HW, Crossley M, Bhoopalan SV, Sharma A, Tisdale JF, Pruett-Miller SM, Cheng Y, Tsai SQ, Liu DR, Weiss MJ, Yen JS. Mayuranathan T, et al. Nat Genet. 2023 Jul;55(7):1210-1220. doi: 10.1038/s41588-023-01434-7. Epub 2023 Jul 3. Nat Genet. 2023. PMID: 37400614 Free PMC article.
CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells.
Brault J, Liu T, Liu S, Lawson A, Choi U, Kozhushko N, Bzhilyanskaya V, Pavel-Dinu M, Meis RJ, Eckhaus MA, Burkett SS, Bosticardo M, Kleinstiver BP, Notarangelo LD, Lazzarotto CR, Tsai SQ, Wu X, Dahl GA, Porteus MH, Malech HL, De Ravin SS. Brault J, et al. Front Immunol. 2023 Jan 4;13:1067417. doi: 10.3389/fimmu.2022.1067417. eCollection 2022. Front Immunol. 2023. PMID: 36685559 Free PMC article.
Base editing as a genetic treatment for spinal muscular atrophy.
Alves CRR, Ha LL, Yaworski R, Lazzarotto CR, Christie KA, Reilly A, Beauvais A, Doll RM, de la Cruz D, Maguire CA, Swoboda KJ, Tsai SQ, Kothary R, Kleinstiver BP. Alves CRR, et al. bioRxiv [Preprint]. 2023 Jan 21:2023.01.20.524978. doi: 10.1101/2023.01.20.524978. bioRxiv. 2023. PMID: 36711797 Free PMC article. Preprint.
CHANGE-seq reveals genetic and epigenetic effects on CRISPR-Cas9 genome-wide activity.
Lazzarotto CR, Malinin NL, Li Y, Zhang R, Yang Y, Lee G, Cowley E, He Y, Lan X, Jividen K, Katta V, Kolmakova NG, Petersen CT, Qi Q, Strelcov E, Maragh S, Krenciute G, Ma J, Cheng Y, Tsai SQ. Lazzarotto CR, et al. Nat Biotechnol. 2020 Nov;38(11):1317-1327. doi: 10.1038/s41587-020-0555-7. Epub 2020 Jun 15. Nat Biotechnol. 2020. PMID: 32541958 Free PMC article.
Base editing of haematopoietic stem cells rescues sickle cell disease in mice.
Newby GA, Yen JS, Woodard KJ, Mayuranathan T, Lazzarotto CR, Li Y, Sheppard-Tillman H, Porter SN, Yao Y, Mayberry K, Everette KA, Jang Y, Podracky CJ, Thaman E, Lechauve C, Sharma A, Henderson JM, Richter MF, Zhao KT, Miller SM, Wang T, Koblan LW, McCaffrey AP, Tisdale JF, Kalfa TA, Pruett-Miller SM, Tsai SQ, Weiss MJ, Liu DR. Newby GA, et al. Nature. 2021 Jul;595(7866):295-302. doi: 10.1038/s41586-021-03609-w. Epub 2021 Jun 2. Nature. 2021. PMID: 34079130 Free PMC article.
12 results