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Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells

Precision gene editing in primary hematopoietic stem and progenitor cells (HSPCs) would facilitate both curative treatments for monogenic disorders as well as disease modelling. Precise efficiencies even with the CRISPR/Cas system, however, remain limited. Through an optimization of guide RNA delive...

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Principais autores: Fanny-Mei Cloarec-Ung, Jamie Beaulieu, Arunan Suthananthan, Bernhard Lehnertz, Guy Sauvageau, Hilary M Sheppard, David JHF Knapp
Formato: Artigo
Idioma:Inglês
Publicado em: eLife Sciences Publications Ltd 2024-06-01
coleção:eLife
Assuntos:
stem cell
hematopoiesis
genome editing
CRISPR
hematopoietic stem cell
leukemia
Acesso em linha:https://elifesciences.org/articles/91288
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