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Author Spotlight: Cardiac Cell Transgenesis for Rapid Gene Screening
Published on: May 24, 2024
Precisely Defined Polymers for Efficient Gene Delivery
Dongsheng He1,2, Hao Lin3,4, Yinglan Yu3,4
1Department of Pharmaceutics, Center for Research Development and Evaluation of Pharmaceutical Excipients and Generic Drugs, School of Pharmacy, China Pharmaceutical University, Nanjing, 210009, People's Republic of China. Dongshenghe@cpu.edu.cn.
Abstract:
Gene therapy requires successful delivery of therapeutic nucleic acids into target cells; thus, efficient and safe gene delivery carriers are crucial to its success. Although many polymeric materials have shown their potential as effective nucleic acid carriers, the inherent heterogeneity and polydispersity of these polymers hinder their application in clinical studies because of difficulties in their further precise modification, structure-activity relationship study, as well as consistent manufacturing. Therefore, precisely defined polymers, with potential for site-specific optimization according to structure-activity relationship information and highly controllable production, have been extensively investigated. In this review, we focus on the design and development of precisely defined polymers for efficient gene delivery, illustrated with examples including dendrimers, peptide-based polymers, and sequence-defined oligoaminoamide oligomers.
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