Abstract

With the development of genome editing techniques, gene therapy is becoming a viable option for the treatment of a variety of human diseases. Genome editing techniques mainly include zinc-finger nucleases, transcription activators like effector nucleases, and the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein system, and these have led to great advancements in biomedical research and therapeutic applications. However, the efficient delivery of genome editing systems to targeted cells remains a great challenge. Nanomaterials possess several advantages for practical applications, including scalability, ease of functionalization, and chemical and thermal stability. Emerging evidence suggests that nanomaterials, such as lipid-based nanomaterials, polymer nanomaterials, porous nanoparticles, and gold nanoparticles, enable efficient gene editing while protecting the target gene against nuclease degradation, improving DNA stability, and increasing the transformation efficiency and safety of gene therapy. In this review, we first introduce three gene editing tools, especially the development and optimization of the CRISPR system. We then introduce recently reported nanobiomaterials that have been applied to gene therapy and discuss the prospects and remaining challenges. Finally, we preview the future direction of gene editing tools, nanobiomaterial vectors, and gene therapy.

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