PAM-flexible Cas9-mediated base editing of a hemophilia B mutation in induced pluripotent stem cells.

Takafumi HiramotoYuji KashiwakuraMorisada Hayakawa Nemekhbayar BaatartsogtNobuhiko KamoshitaTomoyuki Abe Hiroshi InabaHiroshi NishimasuHideki UosakiYutaka HanazonoOsamu Nureki Tsukasa Ohmori

Published in: Communications medicine (2023)

A base-editing approach utilizing the broad PAM flexibility of SpCas9-NG can provide a solution for the treatment of genetic diseases, including hemophilia B.

Keyphrases

crispr cas
genome editing
induced pluripotent stem cells
solid state
combination therapy
gene expression