Restoration of FVIII expression by targeted gene insertion in the FVIII locus in hemophilia A patient-derived iPSCs

Sung, JJ; Park, CY; Leem, JW; Cho, MS; Kim, DW

Kim, DW (reprint author), Yonsei Univ, Coll Med, Dept Physiol, 50-1 Yonsei Ro, Seoul 03722, South Korea.; Kim, DW (reprint author), Yonsei Univ, Coll Med, Brain Korea 21 PLUS Program Med Sci, 50-1 Yonsei Ro, Seoul 03722, South Korea.; Kim, DW (reprint aut

EXPERIMENTAL AND MOLECULAR MEDICINE, 2019; 51 ():

Abstract

Target-specific genome editing, using engineered nucleases zinc finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), and typ......

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