Gene editing rescue of a novel MPL mutant associated with congenital amegakaryocytic thrombocytopenia

Thrombopoietin (Tpo) and its receptor (Mpl) are the principal regulators of early and late thrombopoiesis and hematopoietic stem cell maintenance. Mutations in MPL can drastically impair its function and be a contributing factor in multiple hematologic malignancies, including congenital amegakaryocy...

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Detalhes bibliográficos
Publicado no:Blood Adv
Main Authors: Cleyrat, Cédric, Girard, Romain, Choi, Eun H., Jeziorski, Éric, Lavabre-Bertrand, Thierry, Hermouet, Sylvie, Carillo, Serge, Wilson, Bridget S.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2017
Assuntos:
Platelets and Thrombopoiesis
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5728092/
https://ncbi.nlm.nih.gov/pubmed/29296828
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2016002915
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