Ultra-deep sequencing validates safety of CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cells

Autor: M. Kyle Cromer, Valentin V. Barsan, Erich Jaeger, Mengchi Wang, Jessica P. Hampton, Feng Chen, Drew Kennedy, Jenny Xiao, Irina Khrebtukova, Ana Granat, Tiffany Truong, Matthew H. Porteus
Jazyk: angličtina
Rok vydání: 2022
Předmět:
Zdroj: Nature Communications, Vol 13, Iss 1, Pp 1-11 (2022)
Druh dokumentu: article
ISSN: 2041-1723
DOI: 10.1038/s41467-022-32233-z
Popis: As CRISPR-based therapies enter the clinic, evaluation of safety remains a critical and active area of study. Here the authors use next generation sequencing to achieve high sequencing depth and demonstrate that clinically relevant delivery of high-fidelity Cas9 to primary HSPCs and ex vivo culture up to 10 days does not introduce or enrich for tumorigenic variants.
Databáze: Directory of Open Access Journals