Open AccessGene Therapy: Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retinoschisin 1 Gene—A Potential Nonviral Therapeutic Solution for X‐Linked Juvenile Retinoschisis (Adv. Sci. 10/2020)
Author(s) -
Chou ShihJie,
Yang Peng,
Ban Qian,
Yang YiPing,
Wang MongLien,
Chien ChianShiu,
Chen ShihJen,
Sun Na,
Zhu Yazhen,
Liu Hongtao,
Hui Wenqiao,
Lin TaiChi,
Wang Fang,
Zhang Ryan Yue,
Nguyen Viet Q.,
Liu Wenfei,
Chen Mengxiang,
Jonas Steve J.,
Weiss Paul S.,
Tseng HsianRong,
Chiou ShihHwa
Publication year - 2020
Publication title -
advanced science
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 5.388
H-Index - 100
ISSN - 2198-3844
DOI - 10.1002/advs.202070054
Subject(s) - crispr , cas9 , subgenomic mrna , genetic enhancement , gene , biology , genome editing , in vivo , microbiology and biotechnology , genetics
In article number 1903432, Shih‐Hwa Chiou, Hsian‐Rong Tseng, Paul S. Weiss, and co‐workers introduce an in vivo CRISPR/Cas9‐mediated knockin approach using two supramolecular nanoparticle (SMNP) vectors. By intravitreally injecting the two SMNP vectors into the mouse eyes, the RS1/GFP gene is knocked into the Rosa26 site in mice retinas, offering a revolutionarily curative therapeutic solution for X‐linked juvenile retinoschisis.