Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates.
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| Title: | Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates. |
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| Authors: | 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.; Alimentary Pharmabiotic Centre Microbiome Ireland, University College Cork, Cork T12 K8AF, Ireland., Bao G; Department of Bioengineering, Rice University, Houston, TX 77251, USA., Adair JE; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.; Department of Medicine, University of Washington, Seattle, WA 98195, USA., Peterson CW; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA., Rawlings DJ; Seattle Children's Research Institute, Seattle, WA 98101, USA.; Department of Pediatrics, University of Washington, Seattle, WA 98195, USA.; Department of Immunology, University of Washington, Seattle, WA 98195, USA., Scharenberg AM; Seattle Children's Research Institute, Seattle, WA 98101, USA.; Department of Pediatrics, University of Washington, Seattle, WA 98195, USA.; Department of Immunology, University of Washington, Seattle, WA 98195, USA.; Casebia Therapeutics, Cambridge, MA 02139, USA., Kiem HP; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA. hkiem@fredhutch.org.; Department of Medicine, University of Washington, Seattle, WA 98195, USA. |
| Source: | Science translational medicine [Sci Transl Med] 2019 Jul 31; Vol. 11 (503). |
| Publication Type: | Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1946-6242 |
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| DOI: | 10.1126/scitranslmed.aaw3768 |
