Safe and Effective

Anemia, Sickle Cell Cell Differentiation Gene Editing Genetic Therapy Hematopoietic Stem Cells Humans
gene editing in vivo sickle cell disease targeting

Journal

Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950

Informations de publication

Date de publication:
01 2021
Historique:
pubmed: 12 1 2021
medline: 14 1 2022
entrez: 11 1 2021
Statut: ppublish

Résumé

On May 11, 2020, the National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation (Gates Foundation) held an exploratory expert scientific roundtable to inform an NIH-Gates Foundation collaboration on the development of scalable, sustainable, and accessible HIV and sickle cell disease (SCD) therapies based on

Identifiants

DOI: 10.1089/hum.2020.263 PMID: 33427035 PMC: PMC8917879
pubmed: 33427035
doi: 10.1089/hum.2020.263
pmc: PMC8917879
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

31-42

Subventions

Organisme : NHLBI NIH HHS
ID : R01 HL130040
Pays : United States
Organisme : NHLBI NIH HHS
ID : R01 HL141781
Pays : United States

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Auteurs

Paula Cannon (P)

Keck School of Medicine, University of Southern California, Los Angeles, California, USA.

Aravind Asokan (A)

Department of Surgery, Duke University School of Medicine, Durham, North Carolina, USA.

Agnieszka Czechowicz (A)

Department of Pediatrics.

Paula Hammond (P)

Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA.

Donald B Kohn (DB)

Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA.

Andre Lieber (A)

Department of Medicine, University of Washington, Seattle, Washington, USA.

Punam Malik (P)

Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA.

Peter Marks (P)

U.S. Food and Drug Administration, Silver Spring, Maryland, USA.

Matthew Porteus (M)

Department of Pediatrics.

Els Verhoeyen (E)

CIRI, Université de Lyon, INSERM, CNRS, ENS de Lyon, Lyon, France.
Université de Nice, Nice, France.

Drew Weissman (D)

Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Irving Weissman (I)

Stanford Institute for Stem Cell Biology and Regenerative Medicine; Stanford University School of Medicine, Stanford, California, USA.

Hans-Peter Kiem (HP)

Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

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Classifications MeSH

questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Hémoglobinopathies › Drépanocytose › Safe and Effective
questionsmedicales.fr › Phénomènes physiologiques cellulaires › Différenciation cellulaire › Safe and Effective
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Édition de gène › Safe and Effective
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Safe and Effective
questionsmedicales.fr › Systèmes sanguin et immunitaire › Système hématopoïétique › Cellules de la moelle osseuse › Cellules souches hématopoïétiques › Safe and Effective
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