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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.
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.
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.
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.
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.
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin.
Métais JY, Doerfler PA, Mayuranathan T, Bauer DE, Fowler SC, Hsieh MM, Katta V, Keriwala S, Lazzarotto CR, Luk K, Neel MD, Perry SS, Peters ST, Porter SN, Ryu BY, Sharma A, Shea D, Tisdale JF, Uchida N, Wolfe SA, Woodard KJ, Wu Y, Yao Y, Zeng J, Pruett-Miller S, Tsai SQ, Weiss MJ. Métais JY, et al. Blood Adv. 2019 Nov 12;3(21):3379-3392. doi: 10.1182/bloodadvances.2019000820. Blood Adv. 2019. PMID: 31698466 Free PMC article.
Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease.
Katta V, O'Keefe K, Li Y, Mayuranathan T, Lazzarotto CR, Wood RK, Levine RM, Powers A, Mayberry K, Manquen G, Yao Y, Zhang J, Jang Y, Nimmagadda N, Dempsey EA, Lee G, Uchida N, Cheng Y, Fazio F, Lockey T, Meagher M, Sharma A, Tisdale JF, Zhou S, Yen JS, Weiss MJ, Tsai SQ. Katta V, et al. Mol Ther. 2024 Oct 2;32(10):3433-3452. doi: 10.1016/j.ymthe.2024.07.022. Epub 2024 Jul 31. Mol Ther. 2024. PMID: 39086133 Free PMC article.
16 results