Gene therapy for inborn errors of immunity: Base editing comes into play.
Cell
; 186(7): 1302-1304, 2023 03 30.
Article
en En
| MEDLINE
| ID: mdl-37001495
ABSTRACT
CRISPR-Cas9-based base editing allows precise base editing to achieve conversion of adenosine to guanine or cytosine to thymidine. In this issue of Cell, McAuley et al. use adenine base editing to correct a single base-pair mutation causing human CD3δ deficiency, demonstrating superior efficiency of genetic correction with reduced undesired genetic alterations compared with standard CRISPR-Cas9 editing.
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Sistemas CRISPR-Cas
/
Edición Génica
/
Enfermedades del Sistema Inmune
Límite:
Humans
Idioma:
En
Año:
2023
Tipo del documento:
Article