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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.
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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  Data: Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates.
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  Data: <searchLink fieldCode="AU" term="%22Humbert+O%22">Humbert O</searchLink>; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Radtke+S%22">Radtke S</searchLink>; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Samuelson+C%22">Samuelson C</searchLink>; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Carrillo+RR%22">Carrillo RR</searchLink>; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Perez+AM%22">Perez AM</searchLink>; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Reddy+SS%22">Reddy SS</searchLink>; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Lux+C%22">Lux C</searchLink>; Seattle Children's Research Institute, Seattle, WA 98101, USA.<br /><searchLink fieldCode="AU" term="%22Pattabhi+S%22">Pattabhi S</searchLink>; Seattle Children's Research Institute, Seattle, WA 98101, USA.<br /><searchLink fieldCode="AU" term="%22Schefter+LE%22">Schefter LE</searchLink>; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Negre+O%22">Negre O</searchLink>; Bluebird Bio Inc., Cambridge, MA 02142, USA.<br /><searchLink fieldCode="AU" term="%22Lee+CM%22">Lee CM</searchLink>; Department of Bioengineering, Rice University, Houston, TX 77251, USA.; Alimentary Pharmabiotic Centre Microbiome Ireland, University College Cork, Cork T12 K8AF, Ireland.<br /><searchLink fieldCode="AU" term="%22Bao+G%22">Bao G</searchLink>; Department of Bioengineering, Rice University, Houston, TX 77251, USA.<br /><searchLink fieldCode="AU" term="%22Adair+JE%22">Adair JE</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Peterson+CW%22">Peterson CW</searchLink>; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.<br /><searchLink fieldCode="AU" term="%22Rawlings+DJ%22">Rawlings DJ</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Scharenberg+AM%22">Scharenberg AM</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Kiem+HP%22">Kiem HP</searchLink>; 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.
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  Data: <searchLink fieldCode="JN" term="%22101505086%22">Science translational medicine</searchLink> [Sci Transl Med] 2019 Jul 31; Vol. 11 (503).
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22American+Association+for+the+Advancement+of+Science%22">American Association for the Advancement of Science </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101505086 <i>Publication Model: </i>Print <i>Cited Medium: </i>Internet <i>ISSN: </i>1946-6242 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2219466234%22">19466234 </searchLink><i>NLM ISO Abbreviation: </i>Sci Transl Med <i>Subsets: </i>MEDLINE
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        Text: English
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              Text: 2019 Jul 31
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