Ontology highlight
ABSTRACT:
SUBMITTER: Park CY
PROVIDER: S-EPMC6565751 | biostudies-literature | 2019 Jun
REPOSITORIES: biostudies-literature
Park Chul-Yong CY Sung Jin Jea JJ Cho Sung-Rae SR Kim Jongwan J Kim Dong-Wook DW
Stem cell reports 20190516 6
Hemophilia A (HA) is caused by genetic mutations in the blood coagulation factor VIII (FVIII) gene. Genome-editing approaches can be used to target the mutated site itself in patient-derived induced pluripotent stem cells (iPSCs). However, these approaches can be hampered by difficulty in preparing thousands of editing platforms for each corresponding variant found in HA patients. Here, we report a universal approach to correct the various mutations in HA patient iPSCs by the targeted insertion ...[more]