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CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, Foell J, de la Fuente J, Grupp S, Handgretinger R, Ho TW, Kattamis A, Kernytsky A, Lekstrom-Himes J, Li AM, Locatelli F, Mapara MY, de Montalembert M, Rondelli D, Sharma A, Sheth S, Soni S, Steinberg MH, Wall D, Yen A, Corbacioglu S. Frangoul H, et al. Among authors: sharma a. N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5. N Engl J Med. 2021. PMID: 33283989 Clinical Trial.
Germline Gene Editing for Sickle Cell Disease.
Sharma A, Bhakta N, Johnson LM. Sharma A, et al. Am J Bioeth. 2020 Aug;20(8):46-49. doi: 10.1080/15265161.2020.1781970. Am J Bioeth. 2020. PMID: 32804058 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.
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.
A polygenic score for acute vaso-occlusive pain in pediatric sickle cell disease.
Rampersaud E, Kang G, Palmer LE, Rashkin SR, Wang S, Bi W, Alberts NM, Anghelescu D, Barton M, Birch K, Boulos N, Brandow AM, Brooke RJ, Chang TC, Chen W, Cheng Y, Ding J, Easton J, Hodges JR, Kanne CK, Levy S, Mulder H, Patel AP, Puri L, Rosencrance C, Rusch M, Sapkota Y, Sioson E, Sharma A, Tang X, Thrasher A, Wang W, Yao Y, Yasui Y, Yergeau D, Hankins JS, Sheehan VA, Downing JR, Estepp JH, Zhang J, DeBaun M, Wu G, Weiss MJ. Rampersaud E, et al. Among authors: sharma a. Blood Adv. 2021 Jul 27;5(14):2839-2851. doi: 10.1182/bloodadvances.2021004634. Blood Adv. 2021. PMID: 34283174 Free PMC article.
CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease.
Sharma A, Boelens JJ, Cancio M, Hankins JS, Bhad P, Azizy M, Lewandowski A, Zhao X, Chitnis S, Peddinti R, Zheng Y, Kapoor N, Ciceri F, Maclachlan T, Yang Y, Liu Y, Yuan J, Naumann U, Yu VWC, Stevenson SC, De Vita S, LaBelle JL. Sharma A, et al. N Engl J Med. 2023 Aug 31;389(9):820-832. doi: 10.1056/NEJMoa2215643. N Engl J Med. 2023. PMID: 37646679 Clinical Trial.
Exagamglogene Autotemcel for Severe Sickle Cell Disease.
Frangoul H, Locatelli F, Sharma A, Bhatia M, Mapara M, Molinari L, Wall D, Liem RI, Telfer P, Shah AJ, Cavazzana M, Corbacioglu S, Rondelli D, Meisel R, Dedeken L, Lobitz S, de Montalembert M, Steinberg MH, Walters MC, Eckrich MJ, Imren S, Bower L, Simard C, Zhou W, Xuan F, Morrow PK, Hobbs WE, Grupp SA; CLIMB SCD-121 Study Group. Frangoul H, et al. Among authors: sharma a. N Engl J Med. 2024 May 9;390(18):1649-1662. doi: 10.1056/NEJMoa2309676. Epub 2024 Apr 24. N Engl J Med. 2024. PMID: 38661449 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: sharma a. Blood Adv. 2019 Nov 12;3(21):3379-3392. doi: 10.1182/bloodadvances.2019000820. Blood Adv. 2019. PMID: 31698466 Free PMC article.
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