CRISPR-targeted genome editing of human induced pluripotent stem cell-derived hepatocytes for the treatment of Wilson's disease.
JHEP Rep
; 4(1): 100389, 2022 Jan.
Article
en En
| MEDLINE
| ID: mdl-34877514
AFP, alpha-fetoprotein; ALB, albumin; ATP7B, ATPase copper transporting beta; ATPase copper transporting beta polypeptide (ATP7B); Clustered regularly interspaced palindromic repeats (CRISPR)/Cas9; EB, embryoid body; RFLP, restriction fragment length polymorphism; Single-stranded Oligodeoxynucleotide (ssODN); TGN, trans-Golgi network; WD, Wilson's disease; Wilson's disease; cell therapy; gene correction; iHep(s), iPSC-derived hepatocyte(s); iPSC, induced pluripotent stem cell; iPSC-derived hepatocytes (iHeps); induced pluripotent stem cell (iPSC); sgRNA, single guide RNA; ssODN, single-stranded oligodeoxynucleotide
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Tipo de estudio:
Prognostic_studies
Idioma:
En
Revista:
JHEP Rep
Año:
2022
Tipo del documento:
Article
País de afiliación:
China
Pais de publicación:
Países Bajos