Manchanda Deeksha, Kumar Sunil, Makhija Manish
Department of Pharmaceutical Sciences, Indira Gandhi University, Meerpur, Rewari - 123401, India.
Curr Gene Ther. 2025 Mar 10. doi: 10.2174/0115665232369121250307075817.
The advent of CRISPR/Cas gene-editing technology has revolutionized molecular biology, offering unprecedented precision and potential in treating genetic disorders, cancers, and other complex diseases. However, for CRISPR/Cas to be truly effective in clinical settings, one of the most significant challenges lies in the delivery of the CRISPR components, including guide RNA (gRNA) and Cas protein, into specific cells or tissues. Safe, targeted, and efficient delivery remains a critical bottleneck. Viral vectors, lipid nanoparticles, and synthetic polymers have been explored, but they come with limitations, such as immunogenicity, toxicity, and limited delivery capacity. Polysaccharide-based delivery systems, with their natural origin, biocompatibility, and versatile chemical properties, offer a promising alternative that could address these delivery challenges while advancing the pharmaceutical applications of CRISPR/Cas gene therapy.
CRISPR/Cas基因编辑技术的出现彻底改变了分子生物学，在治疗遗传疾病、癌症和其他复杂疾病方面提供了前所未有的精准度和潜力。然而，要使CRISPR/Cas在临床环境中真正有效，最重大的挑战之一在于将CRISPR组件，包括引导RNA（gRNA）和Cas蛋白，递送至特定的细胞或组织中。安全、靶向且高效的递送仍然是一个关键瓶颈。人们已经探索了病毒载体、脂质纳米颗粒和合成聚合物，但它们存在局限性，如免疫原性、毒性和递送能力有限。基于多糖的递送系统，因其天然来源、生物相容性和多样的化学性质，提供了一种有前景的替代方案，有望解决这些递送挑战，同时推动CRISPR/Cas基因治疗的药物应用。
