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Cyclization-enhanced poly(β-amino ester)s vectors for efficient CRISPR gene editing therapy

基因传递 转染 基因 清脆的 报告基因 基因组编辑 质粒 遗传增强 DNA 体外 化学 基因表达 计算生物学 生物 分子生物学 生物物理学 生物化学
作者
Xianqing Wang,Yinghao Li,A Sigen,Jing Wang,Xi Wang,Zhonglei He,Irene Lara‐Sáez,Ming Li,Wenxin Wang
出处
期刊:Journal of Controlled Release [Elsevier]
卷期号:368: 444-452 被引量:1
标识
DOI:10.1016/j.jconrel.2024.02.032
摘要

Among non-viral gene delivery vectors, poly(β-amino ester)s (PAEs) are one of the most versatile candidates because of their wide monomer availability, high polymer flexibility, and superior gene transfection performance both in vitro and in vivo. Over two decades, PAEs have evolved from linear to highly branched structures, significantly enhancing gene delivery efficacy. Building on the proven efficient sets of monomers in highly branched PAEs (HPAEs), this work introduced a new class of cyclic PAEs (CPAEs) constructed via an A2 + B4 + C2 cyclization synthesis strategy and identified their markedly improved gene transfection capabilities in gene delivery applications. Two sets of cyclic PAEs (CPAEs) with rings of different sizes and topologies were obtained. Their chemical structures were confirmed via two-dimensional nuclear magnetic resonance and the photoluminescence phenomena, and their DNA delivery behaviours were investigated and compared with the HPAE counterparts. In vitro assessments demonstrated that the CPAEs with a macrocyclic architecture (MCPAEs), significantly enhanced DNA intracellular uptake and facilitated efficient gene expression while maintaining perfect biocompatibility. The top-performance MCPAEs have been further employed to deliver a plasmid coding dual single guide RNA-guided CRISPR-Cas9 machinery to delete COL7A1 exon 80 containing the c.6527dupC mutation. In recessive dystrophic epidermolysis bullosa (RDEB) patient-derived epidermal keratinocytes, MCPAEs facilitated the CRISPR plasmid delivery and achieved efficient targeted gene editing in multiple colonies.
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