CRISPR/Cas9-mediated ELANE knockout enables neutrophilic maturation of primary hematopoietic stem and progenitor cells and induced pluripotent stem cells of severe congenital neutropenia patients

A Autosomal-dominant ELANE mutations are the most common cause of severe congenital neutropenia. Although the majority of congenital neutropenia patients respond to daily granulocyte colony stimulating factor, approximately 15 % do not respond to this cytokine at doses up to 50 μg/kg/day and approxi...

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Publicado no:Haematologica
Main Authors: Nasri, Masoud, Ritter, Malte, Mir, Perihan, Dannenmann, Benjamin, Aghaallaei, Narges, Amend, Diana, Makaryan, Vahagn, Xu, Yun, Fletcher, Breanna, Bernhard, Regine, Steiert, Ingeborg, Hahnel, Karin, Berger, Jürgen, Koch, Iris, Sailer, Brigitte, Hipp, Katharina, Zeidler, Cornelia, Klimiankou, Maksim, Bajoghli, Baubak, Dale, David C., Welte, Karl, Skokowa, Julia
Formato: Artigo
Idioma:Inglês
Publicado em: Ferrata Storti Foundation 2020
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Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7049355/
https://ncbi.nlm.nih.gov/pubmed/31248972
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3324/haematol.2019.221804
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