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Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates.
Humbert, Olivier; Radtke, Stefan; Samuelson, Clare; Carrillo, Ray R; Perez, Anai M; Reddy, Sowmya S; Lux, Christopher; Pattabhi, Sowmya; Schefter, Lauren E; Negre, Olivier; Lee, Ciaran M; Bao, Gang; Adair, Jennifer E; Peterson, Christopher W; Rawlings, David J; Scharenberg, Andrew M; Kiem, Hans-Peter.
Afiliación
  • Humbert O; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
  • Radtke S; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
  • Samuelson C; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
  • Carrillo RR; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
  • Perez AM; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
  • Reddy SS; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
  • Lux C; Seattle Children's Research Institute, Seattle, WA 98101, USA.
  • Pattabhi S; Seattle Children's Research Institute, Seattle, WA 98101, USA.
  • Schefter LE; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
  • Negre O; Bluebird Bio Inc., Cambridge, MA 02142, USA.
  • Lee CM; Department of Bioengineering, Rice University, Houston, TX 77251, USA.
  • Bao G; Alimentary Pharmabiotic Centre Microbiome Ireland, University College Cork, Cork T12 K8AF, Ireland.
  • Adair JE; Department of Bioengineering, Rice University, Houston, TX 77251, USA.
  • Peterson CW; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
  • Rawlings DJ; Department of Medicine, University of Washington, Seattle, WA 98195, USA.
  • Scharenberg AM; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
  • Kiem HP; Seattle Children's Research Institute, Seattle, WA 98101, USA.
Sci Transl Med ; 11(503)2019 07 31.
Article en En | MEDLINE | ID: mdl-31366580
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Hemoglobina Fetal / Células Madre Hematopoyéticas / Sistemas CRISPR-Cas Límite: Animals / Humans Idioma: En Revista: Sci Transl Med Asunto de la revista: CIENCIA / MEDICINA Año: 2019 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Hemoglobina Fetal / Células Madre Hematopoyéticas / Sistemas CRISPR-Cas Límite: Animals / Humans Idioma: En Revista: Sci Transl Med Asunto de la revista: CIENCIA / MEDICINA Año: 2019 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos