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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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Dades bibliogràfiques
Publicat a:Blood Adv
Autors principals: Cleyrat, Cédric, Girard, Romain, Choi, Eun H., Jeziorski, Éric, Lavabre-Bertrand, Thierry, Hermouet, Sylvie, Carillo, Serge, Wilson, Bridget S.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Hematology 2017
Matèries:
Accés en línia: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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