Your browser doesn't support javascript.
loading
Mostrar: 20 | 50 | 100
Resultados 1 - 1 de 1
Filtrar
Más filtros

Bases de datos
Tipo del documento
País de afiliación
Intervalo de año de publicación
1.
Cell Stem Cell ; 26(4): 503-510.e7, 2020 04 02.
Artículo en Inglés | MEDLINE | ID: mdl-32084388

RESUMEN

Adenine base editing (ABE) enables enzymatic conversion from A-T into G-C base pairs. ABE holds promise for clinical application, as it does not depend on the introduction of double-strand breaks, contrary to conventional CRISPR/Cas9-mediated genome engineering. Here, we describe a cystic fibrosis (CF) intestinal organoid biobank, representing 664 patients, of which ~20% can theoretically be repaired by ABE. We apply SpCas9-ABE (PAM recognition sequence: NGG) and xCas9-ABE (PAM recognition sequence: NGN) on four selected CF organoid samples. Genetic and functional repair was obtained in all four cases, while whole-genome sequencing (WGS) of corrected lines of two patients did not detect off-target mutations. These observations exemplify the value of large, patient-derived organoid biobanks representing hereditary disease and indicate that ABE may be safely applied in human cells.


Asunto(s)
Repeticiones Palindrómicas Cortas Agrupadas y Regularmente Espaciadas , Fibrosis Quística , Adenina , Bancos de Muestras Biológicas , Proteína 9 Asociada a CRISPR/genética , Sistemas CRISPR-Cas/genética , Codón sin Sentido , Fibrosis Quística/genética , Edición Génica , Humanos , Organoides/metabolismo
SELECCIÓN DE REFERENCIAS
DETALLE DE LA BÚSQUEDA