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Page 1
Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease.
Leonard A, Sharma A, Uchida N, Stroncek D, Panch SR, West K, Molloy E, Hughes TE, Hauffe S, Taylor T, Fitzhugh C, Hankins JS, Wilson M, Tsai SQ, Weiss MJ, Hsieh M, Tisdale JF. Leonard A, et al. Among authors: tsai sq. Blood Adv. 2021 May 11;5(9):2403-2411. doi: 10.1182/bloodadvances.2021004232. Blood Adv. 2021. PMID: 33956057 Free PMC article. Clinical Trial.
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.
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.
Safe and efficient peripheral blood stem cell collection in patients with sickle cell disease using plerixafor.
Uchida N, Leonard A, Stroncek D, Panch SR, West K, Molloy E, Hughes TE, Hauffe S, Taylor T, Fitzhugh C, Hankins JS, Wilson M, Sharma A, Tsai SQ, Weiss MJ, Hsieh M, Tisdale JF. Uchida N, et al. Among authors: tsai sq. Haematologica. 2020 Oct 1;105(10):e497. doi: 10.3324/haematol.2019.236182. Haematologica. 2020. PMID: 33054091 Free PMC article. No abstract available.
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.
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. Among authors: tsai sq. 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.
BCL11A +58/+55 enhancer-editing facilitates HSPC engraftment and HbF induction in rhesus macaques conditioned with a CD45 antibody-drug conjugate.
Demirci S, Zeng J, Palchaudhuri R, Wu C, Abraham DM, Hayal TB, Essawi K, Nguyen MA, Stasula U, Chu R, Leonard A, Porter SN, Khan MBN, Hinojosa G, Uchida N, Hong S, Lazzarotto CR, Neri NR, da Silva LF, Pellin D, Verma A, Lanieri L, Bhat A, Hammond K, Tate T, Maitland SA, Sheikhsaran F, Bonifacino AC, Krouse AE, Linde NS, Engels T, Golomb J, Tsai SQ, Pruett-Miller SM, Scadden DT, Dunbar CE, Wolfe SA, Donahue RE, Olson LM, Bauer DE, Tisdale JF. Demirci S, et al. Among authors: tsai sq. Cell Stem Cell. 2024 Dec 5:S1934-5909(24)00376-X. doi: 10.1016/j.stem.2024.10.014. Online ahead of print. Cell Stem Cell. 2024. PMID: 39642886
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.
66 results