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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: sharma a. 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.
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: sharma a. Blood Adv. 2019 Nov 12;3(21):3379-3392. doi: 10.1182/bloodadvances.2019000820. Blood Adv. 2019. PMID: 31698466 Free PMC article.
Improved survival rate in T-cell depleted haploidentical hematopoietic cell transplantation over the last 15 years at a single institution.
Mamcarz E, Madden R, Qudeimat A, Srinivasan A, Talleur A, Sharma A, Suliman A, Maron G, Sunkara A, Kang G, Leung W, Gottschalk S, Triplett BM. Mamcarz E, et al. Among authors: sharma a. Bone Marrow Transplant. 2020 May;55(5):929-938. doi: 10.1038/s41409-019-0750-7. Epub 2019 Nov 18. Bone Marrow Transplant. 2020. PMID: 31740766 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: sharma a. Haematologica. 2020 Oct 1;105(10):e497. doi: 10.3324/haematol.2019.236182. Haematologica. 2020. PMID: 33054091 Free PMC article. No abstract available.
Genetic therapies for the first molecular disease.
Doerfler PA, Sharma A, Porter JS, Zheng Y, Tisdale JF, Weiss MJ. Doerfler PA, et al. Among authors: sharma a. J Clin Invest. 2021 Apr 15;131(8):e146394. doi: 10.1172/JCI146394. J Clin Invest. 2021. PMID: 33855970 Free PMC article. Review.
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: sharma a. Blood Adv. 2021 May 11;5(9):2403-2411. doi: 10.1182/bloodadvances.2021004232. Blood Adv. 2021. PMID: 33956057 Free PMC article. Clinical Trial.
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: sharma a. 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.
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