CRISPR-Cas9-Mediated ELANE Mutation Correction in Hematopoietic Stem and Progenitor Cells to Treat Severe Congenital Neutropenia

Severe congenital neutropenia (SCN) is a monogenic disorder. SCN patients are prone to recurrent life-threatening infections. The main causes of SCN are autosomal dominant mutations in the ELANE gene that lead to a block in neutrophil differentiation. In this study, we use CRISPR-Cas9 ribonucleoprot...

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Publicat a:Mol Ther
Autors principals: Tran, Ngoc Tung, Graf, Robin, Wulf-Goldenberg, Annika, Stecklum, Maria, Strauß, Gabriele, Kühn, Ralf, Kocks, Christine, Rajewsky, Klaus, Chu, Van Trung
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2020
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7704744/
https://ncbi.nlm.nih.gov/pubmed/32822592
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.08.004
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