Effective therapies for sickle cell disease: are we there yet?

Crossley, Merlin; Christakopoulos, Georgios E; Weiss, Mitchell J

Crossley, Merlin; Christakopoulos, Georgios E; Weiss, Mitchell J.

Afiliación

Crossley M; School of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, Australia 2052. Electronic address: m.crossley@unsw.edu.au.
Christakopoulos GE; Department of Oncology, St Jude Children's Research Hospital, Memphis, TN 38105, USA.
Weiss MJ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.

Trends Genet ; 38(12): 1284-1298, 2022 12.

Article en En | MEDLINE | ID: mdl-35934593

Asunto(s)

Anemia de Células Falciformes; Trasplante de Células Madre Hematopoyéticas; Humanos; Anemia de Células Falciformes/genética; Anemia de Células Falciformes/terapia; Edición Génica/métodos; Células Madre Hematopoyéticas; Terapia Genética

Palabras clave

CRISPR; base editing; gene editing; gene therapy; sickle cell disease; ß-hemoglobinopathies; ß-thalassemia

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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Trasplante de Células Madre Hematopoyéticas / Anemia de Células Falciformes Límite: Humans Idioma: En Año: 2022 Tipo del documento: Article

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