Baig Mirza Muhammad Faran Ashraf, Chien Wai Tong, Chair Sek Ying
Croucher Laboratory for Human Genomics, Asia Pacific Genetic and Genomic Nursing Center, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China.
Biomater Sci. 2025 Aug 19;13(17):4597-4638. doi: 10.1039/d5bm00711a.
The integration strategies of CRISPR-Cas gene editing systems with nanotechnological approaches have achieved synergistic effects in targeting genes; correcting genetic disorders; and treating, sensing, and diagnosing a variety of cancers and metabolic, immunological, and complex infectious diseases-all having connectivity with distinct genetic cues and mutations. Numerous recent studies have demonstrated the use of the nano-scale properties of nanomaterials to tremendously improve the genomic-editing efficiencies of CRISPR/Cas systems for achieving 50% enhanced bioavailability, improved cell targetability, and gene-level specificity while minimizing immunogenicity, compared with conventional/ordinary delivery techniques. Thus, nano-delivery methods utilizing the unique properties of nanomaterials, molecular interactions, biocompatibility, targeted cellular uptake, and nuclear delivery capability effectively overcame the challenges of inefficient biomolecular delivery, and off-target effects were effectively overcome. Nano -carriers made up of materials such as DNA lattices, lipids, dendrimers, polymers, peptides, and metals (gold, silver, .) that were explored for facilitating the precise delivery of CRISPR/Cas components, sensing biomolecules, and diagnostic purposes are discussed in this review report. The ability of DNA scaffold materials to incorporate nano-CRISPR systems, to sense biomolecules, and for targeted cellular delivery of payloads (, Cas9, Cas12, Cas13, and Cas14 proteins and single-guide RNAs (sgRNAs)) maximized gene targeting and improved therapeutic outcomes while achieving up to 90% efficiency compared with common/trivial delivery methods.
CRISPR-Cas基因编辑系统与纳米技术方法的整合策略在基因靶向、纠正遗传疾病以及治疗、传感和诊断多种癌症、代谢性疾病、免疫性疾病和复杂感染性疾病方面均取得了协同效应，所有这些疾病都与独特的遗传线索和突变相关。最近的大量研究表明，与传统/普通递送技术相比，利用纳米材料的纳米级特性可极大提高CRISPR/Cas系统的基因组编辑效率，实现生物利用度提高50%、细胞靶向性改善以及基因水平特异性增强，同时将免疫原性降至最低。因此，利用纳米材料独特性质、分子相互作用、生物相容性、靶向细胞摄取和核递送能力的纳米递送方法有效克服了生物分子递送效率低下的挑战，并有效克服了脱靶效应。本综述报告讨论了由DNA晶格、脂质、树枝状大分子、聚合物、肽和金属（金、银等）等材料制成的纳米载体，这些纳米载体用于促进CRISPR/Cas组件的精确递送、传感生物分子和诊断目的。与普通/常规递送方法相比，DNA支架材料整合纳米CRISPR系统、传感生物分子以及靶向细胞递送有效载荷（如Cas9、Cas12、Cas13和Cas14蛋白以及单向导RNA（sgRNA））的能力可最大化基因靶向并改善治疗效果，同时实现高达90%的效率。
