Cationic polymeric nanoformulation: Recent advances in material design for CRISPR/Cas9 gene therapy

Publication date: Available online 10 December 2019Source: Progress in Natural Science: Materials InternationalAuthor(s): Kaifeng Chen, Shan Jiang, Yun Hong, Zibiao Li, Yun-Long Wu, Caisheng WuAbstractCRISPR/Cas9 (clustered regularly interspaced short palindromic repeat/clustered regularly interspaced short palindromic repeat associated proteins 9) gene editing platform is a promising therapeutic tool for genetic disorders, due to its ability to manipulate the pathogenic gene in genomic level and to easily target specific gene by manipulating single-guide RNA. However, its successful delivery remains a challenge. Up to now, great efforts have been made to explore an effective strategy for CRISPR/Cas9 delivery. But among those delivery methods, physical methods are mainly operated on cultured cells thus limited to laboratorial use; viral vectors are hindered by fetal immunogenic and carcinogenic effects thus dubious in clinical application. Therefore, cationic polymeric vectors, with the ability to interact with CRISPR/Cas9 system to form a nanoformulation as a non-viral approach, are attracting increasing attentions, due to advantages such as well protection of cargos, less limitation in payload size, low immunogenicity or carcinogenicity, potential modifications for further functions, and ease in mass production. In this review, the recent discoveries on polymeric vectors utilized in delivery of CRISPR/Cas9 system will be summarized. With emphasis on advanced features of tho...
Source: Progress in Natural Science: Materials International - Category: Materials Science Source Type: research