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...

Descripción completa

Detalles Bibliográficos
Autores principales: Tran, Ngoc Tung, Graf, Robin, Wulf-Goldenberg, Annika, Stecklum, Maria, Strauß, Gabriele, Kühn, Ralf, Kocks, Christine, Rajewsky, Klaus, Chu, Van Trung
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7704744/
https://www.ncbi.nlm.nih.gov/pubmed/32822592
http://dx.doi.org/10.1016/j.ymthe.2020.08.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7704744/
https://www.ncbi.nlm.nih.gov/pubmed/32822592
http://dx.doi.org/10.1016/j.ymthe.2020.08.004

Ejemplares similares