Generation of iPSC line from urine cells of hemophilia A with F8 (p. R814X) mutation.
Stem Cell Res
; 60: 102682, 2022 04.
Article
em En
| MEDLINE
| ID: mdl-35123343
ABSTRACT
The lack of coagulation factor VIII in patient with nonsense mutation hemophilia A leads to varying degrees of bleeding symptoms, and long-term use of alternative therapies can produce inhibitors that affect the efficacy. In this study, human induced pluripotent stem cells (iPSCs) of hemophilia A were generated by reprogramming of urine cells. Human urine cells (HUCs) were isolated by collecting patients' mid-stream urine, and cultured to good state in urine medium. Then, the HUCs were transfected with PEP4-EO2S-ET2K and pCEP4-M2L, and iPSCs were obtained in the medium without trophoblast cells and the composition was determined. Finally, alkaline phosphatase staining, karyotype analysis, immunofluorescence staining and teratoma were used to verify that we successfully reprogrammed hemophilia A-specific human induced pluripotent stem cells from patients' urine cells, providing a safe and effective cell model for the study of molecular mechanism and related treatment of hemophilia.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Células-Tronco Pluripotentes Induzidas
/
Hemofilia A
Tipo de estudo:
Prognostic_studies
Idioma:
En
Revista:
Stem Cell Res
Ano de publicação:
2022
Tipo de documento:
Article