Successful correction of factor V deficiency of patient-derived iPSCs by CRISPR/Cas9-mediated gene editing.

Takayuki NakamuraSatoshi MorishigeHidetoshi OzawaKenji KuboyamaYoshitaka YamasakiShuki OyaMaki YamaguchiKazutoshi AoyamaRitsuko SekiFumihiko MouriKoichi OsakiTakashi OkamuraShinichi MizunoKoji Nagafuji

Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2020)

We successfully repaired the mutation of F5 using the CRISPR/Cas9 and confirmed the recovery of FV activity with gene-corrected iPS-HLCs. Gene-edited iPSCs are promising for elucidating the pathophysiology as well as for a modality of gene therapy.

Keyphrases

crispr cas
genome editing
gene therapy
copy number
genome wide
genome wide identification
induced pluripotent stem cells
gene expression
dna methylation
replacement therapy