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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Principais autores:	Masoud Nasri, Malte Ritter, Perihan Mir, Benjamin Dannenmann, Narges Aghaallaei, Diana Amend, Vahagn Makaryan, Yun Xu, Breanna Fletcher, Regine Bernhard, Ingeborg Steiert, Karin Hahnel, Jürgen Berger, Iris Koch, Brigitte Sailer, Katharina Hipp, Cornelia Zeidler, Maksim Klimiankou, Baubak Bajoghli, David C. Dale, Karl Welte, Julia Skokowa
Format:	Artigo
Jezik:	Inglês
Izdano:	Ferrata Storti Foundation 2020-03-01
Serija:	Haematologica
Online dostop:	https://haematologica.org/article/view/9279
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