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CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease.
Sharma, Akshay; Boelens, Jaap-Jan; Cancio, Maria; Hankins, Jane S; Bhad, Prafulla; Azizy, Marjohn; Lewandowski, Andrew; Zhao, Xiaojun; Chitnis, Shripad; Peddinti, Radhika; Zheng, Yan; Kapoor, Neena; Ciceri, Fabio; Maclachlan, Timothy; Yang, Yi; Liu, Yi; Yuan, Jianping; Naumann, Ulrike; Yu, Vionnie W C; Stevenson, Susan C; De Vita, Serena; LaBelle, James L.
Afiliação
  • Sharma A; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Boelens JJ; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Cancio M; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Hankins JS; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Bhad P; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Azizy M; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Lewandowski A; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Zhao X; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Chitnis S; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Peddinti R; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Zheng Y; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Kapoor N; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Ciceri F; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Maclachlan T; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Yang Y; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Liu Y; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Yuan J; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Naumann U; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Yu VWC; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • Stevenson SC; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • De Vita S; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
  • LaBelle JL; From St. Jude Children's Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University
N Engl J Med ; 389(9): 820-832, 2023 Aug 31.
Article em En | MEDLINE | ID: mdl-37646679
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Hemoglobina Fetal / Transplante de Células-Tronco Hematopoéticas / Eritrócitos / Sistemas CRISPR-Cas / Anemia Falciforme Limite: Animals Idioma: En Revista: N Engl J Med Ano de publicação: 2023 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Hemoglobina Fetal / Transplante de Células-Tronco Hematopoéticas / Eritrócitos / Sistemas CRISPR-Cas / Anemia Falciforme Limite: Animals Idioma: En Revista: N Engl J Med Ano de publicação: 2023 Tipo de documento: Article