The group of Chui Yong PARK at the Stem Cell Institute of Yonsei University reported at the Asia Cell Therapy Society 6th Annual Meeting that the combination of iPS cell therapy with CRISPR/Cas9 genome editing techniques showed some success in a mouse model (significantly prolonged survival period). The factor VIII coding gene was derived from a human hemophilia A patient and modified by genome editing in intron 1 and intron 22.
Lecture at Chui Yong PARK at ACTO meeting on August 20, 2015
Group at Yonsei University sees success in genome editing for repaired factor VIII, mouse experiments