AUTHOR=Chien Chian-Shiu , Chien Yueh , Lin Yi-Ying , Tsai Ping-Hsing , Chou Shih-Jie , Yarmishyn Aliaksandr A. , Rastegari Elham , Wang Ting-Xian , Leu Hsin-Bang , Yang Yi-Ping , Wang Mong-Lien , Jheng Ying-Chun , Lai Henkie Isahwan Ahmad Mulyadi , Ching Lo-Jei , Huo Teh-Ia , Cherng Jong-Yuh , Wang Chien-Ying TITLE=Dual DNA Transfection Using 1,6-Hexanedithiol-Conjugated Maleimide-Functionalized PU-PEI600 For Gene Correction in a Patient iPSC-Derived Fabry Cardiomyopathy Model JOURNAL=Frontiers in Cell and Developmental Biology VOLUME=Volume 9 - 2021 YEAR=2021 URL=https://www.frontiersin.org/journals/cell-and-developmental-biology/articles/10.3389/fcell.2021.634190 DOI=10.3389/fcell.2021.634190 ISSN=2296-634X ABSTRACT=
Non-viral gene delivery holds promises for treating inherited diseases. However, the limited cloning capacity of plasmids may hinder the co-delivery of distinct genes to the transfected cells. Previously, the conjugation of maleimide-functionalized polyurethane grafted with small molecular weight polyethylenimine (PU-PEI600-Mal) using 1,6-hexanedithiol (HDT) could promote the co-delivery and extensive co-expression of two different plasmids in target cells. Herein, we designed HDT-conjugated PU-PEI600-Mal for the simultaneous delivery of CRISPR/Cas9 components to achieve efficient gene correction in the induced pluripotent stem cell (iPSC)-derived model of Fabry cardiomyopathy (FC) harboring