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Page 1
Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia.
Kim MY, Yu KR, Kenderian SS, Ruella M, Chen S, Shin TH, Aljanahi AA, Schreeder D, Klichinsky M, Shestova O, Kozlowski MS, Cummins KD, Shan X, Shestov M, Bagg A, Morrissette JJD, Sekhri P, Lazzarotto CR, Calvo KR, Kuhns DB, Donahue RE, Behbehani GK, Tsai SQ, Dunbar CE, Gill S. Kim MY, et al. Among authors: tsai sq. Cell. 2018 May 31;173(6):1439-1453.e19. doi: 10.1016/j.cell.2018.05.013. Epub 2018 May 31. Cell. 2018. PMID: 29856956 Free PMC article.
Defining CRISPR-Cas9 genome-wide nuclease activities with CIRCLE-seq.
Lazzarotto CR, Nguyen NT, Tang X, Malagon-Lopez J, Guo JA, Aryee MJ, Joung JK, Tsai SQ. Lazzarotto CR, et al. Among authors: tsai sq. Nat Protoc. 2018 Nov;13(11):2615-2642. doi: 10.1038/s41596-018-0055-0. Nat Protoc. 2018. PMID: 30341435 Free PMC article.
Highly efficient therapeutic gene editing of human hematopoietic stem cells.
Wu Y, Zeng J, Roscoe BP, Liu P, Yao Q, Lazzarotto CR, Clement K, Cole MA, Luk K, Baricordi C, Shen AH, Ren C, Esrick EB, Manis JP, Dorfman DM, Williams DA, Biffi A, Brugnara C, Biasco L, Brendel C, Pinello L, Tsai SQ, Wolfe SA, Bauer DE. Wu Y, et al. Among authors: tsai sq. Nat Med. 2019 May;25(5):776-783. doi: 10.1038/s41591-019-0401-y. Epub 2019 Mar 25. Nat Med. 2019. PMID: 30911135 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. Among authors: tsai sq. Blood Adv. 2019 Nov 12;3(21):3379-3392. doi: 10.1182/bloodadvances.2019000820. Blood Adv. 2019. PMID: 31698466 Free PMC article.
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. Among authors: tsai sq. 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.
BCL11A enhancer-edited hematopoietic stem cells persist in rhesus monkeys without toxicity.
Demirci S, Zeng J, Wu Y, Uchida N, Shen AH, Pellin D, Gamer J, Yapundich M, Drysdale C, Bonanno J, Bonifacino AC, Krouse AE, Linde NS, Engels T, Donahue RE, Haro-Mora JJ, Leonard A, Nassehi T, Luk K, Porter SN, Lazzarotto CR, Tsai SQ, Weiss MJ, Pruett-Miller SM, Wolfe SA, Bauer DE, Tisdale JF. Demirci S, et al. Among authors: tsai sq. J Clin Invest. 2020 Dec 1;130(12):6677-6687. doi: 10.1172/JCI140189. J Clin Invest. 2020. PMID: 32897878 Free PMC article.
Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells.
De Ravin SS, Brault J, Meis RJ, Liu S, Li L, Pavel-Dinu M, Lazzarotto CR, Liu T, Koontz SM, Choi U, Sweeney CL, Theobald N, Lee G, Clark AB, Burkett SS, Kleinstiver BP, Porteus MH, Tsai S, Kuhns DB, Dahl GA, Headey S, Wu X, Malech HL. De Ravin SS, et al. Blood. 2021 May 13;137(19):2598-2608. doi: 10.1182/blood.2020008503. Blood. 2021. PMID: 33623984 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. Among authors: tsai sq. 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.
CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes.
Brault J, Liu T, Bello E, Liu S, Sweeney CL, Meis RJ, Koontz S, Corsino C, Choi U, Vayssiere G, Bosticardo M, Dowdell K, Lazzarotto CR, Clark AB, Notarangelo LD, Ravell JC, Lenardo MJ, Kleinstiver BP, Tsai SQ, Wu X, Dahl GA, Malech HL, De Ravin SS. Brault J, et al. Among authors: tsai sq. Blood. 2021 Dec 30;138(26):2768-2780. doi: 10.1182/blood.2021011192. Blood. 2021. PMID: 34086870 Free PMC article.
Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques.
AlJanahi AA, Lazzarotto CR, Chen S, Shin TH, Cordes S, Fan X, Jabara I, Zhou Y, Young DJ, Lee BC, Yu KR, Li Y, Toms B, Tunc I, Hong SG, Truitt LL, Klermund J, Andrieux G, Kim MY, Cathomen T, Gill S, Tsai SQ, Dunbar CE. AlJanahi AA, et al. Among authors: tsai sq. Mol Ther. 2022 Jan 5;30(1):209-222. doi: 10.1016/j.ymthe.2021.06.016. Epub 2021 Jun 24. Mol Ther. 2022. PMID: 34174439 Free PMC article.
64 results