Genome editing in human hematopoietic stem and progenitor cells via CRISPR-Cas9-mediated homology-independent targeted integration

Ex vivo gene correction of hematopoietic stem and progenitor cells (HSPCs) has emerged as a promising therapeutic approach for treatment of inherited human blood disorders. Use of engineered nucleases to target therapeutic transgenes to their endogenous genetic loci addresses many of the limitations...

Descripción completa

Detalles Bibliográficos
Autores principales: Bloomer, Hanan, Smith, Richard H., Hakami, Waleed, Larochelle, Andre
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8058434/
https://www.ncbi.nlm.nih.gov/pubmed/33309880
http://dx.doi.org/10.1016/j.ymthe.2020.12.010

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8058434/
https://www.ncbi.nlm.nih.gov/pubmed/33309880
http://dx.doi.org/10.1016/j.ymthe.2020.12.010

Ejemplares similares