Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retinoschisin 1 Gene—A Potential Nonviral Therapeutic Solution for X‐Linked Juvenile Retinoschisis

The homology‐independent targeted integration (HITI) strategy enables effective CRISPR/Cas9‐mediated knockin of therapeutic genes in nondividing cells in vivo, promising general therapeutic solutions for treating genetic diseases like X‐linked juvenile retinoschisis. Herein, supramolecular nanoparti...

Description complète

Enregistré dans:
Détails bibliographiques
Publié dans:Adv Sci (Weinh)
Auteurs principaux: Chou, Shih‐Jie, Yang, Peng, Ban, Qian, Yang, Yi‐Ping, Wang, Mong‐Lien, Chien, Chian‐Shiu, Chen, Shih‐Jen, Sun, Na, Zhu, Yazhen, Liu, Hongtao, Hui, Wenqiao, Lin, Tai‐Chi, Wang, Fang, Zhang, Ryan Yue, Nguyen, Viet Q., Liu, Wenfei, Chen, Mengxiang, Jonas, Steve J., Weiss, Paul S., Tseng, Hsian‐Rong, Chiou, Shih‐Hwa
Format: Artigo
Langue:Inglês
Publié: John Wiley and Sons Inc. 2020
Sujets:
Communications
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC7237855/
https://ncbi.nlm.nih.gov/pubmed/32440478
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/advs.201903432
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