Jinka Chaitra, Sainath Chithirala, Babu Shyamaladevi, Chennupati Ashok Chakravarthi, Muppidi Lakshmi Prasanna, Krishnan Madhan, Sekar Gayathri, Chinnaiyan Mayilvanan, Andugula Swapna Kumari
Department of Animal Biotechnology, Sri Venkateswara University, Tirupati - 517502.
Syngene International Limited, Biocon park, Bangalore, Karnataka, India.
Bioinformation. 2022 Nov 30;18(11):1081-1086. doi: 10.6026/973206300181081. eCollection 2022.
CRISPR/Cas-9 mediated genome editing has recently emerged as a potential and innovative technology in therapeutic development and biomedical research. Several recent studies have been performed to understand gene modification techniques in obtaining effective ex vivo results. Generally, the disease targets for gene correction will be in specific organs, so understanding the complete potential of genomic treatment methods is essential. From such a perspective, the present review revealed the significant importance of the CRISPR/ Cas9 delivery system. Both the promising gene-editing delivery systems, such as synthetic (non-viral) and viral vector systems are discussed in this review. In addition, this paper attempted to summarize the tissue-specific and organ-specific mRNA delivery systems that provide possible research information for future researchers. Further, the major challenges of the CRISPR/Cas9 system, such as off-target delivery, immunogenicity, and limited packaging, were also elucidated. Accordingly, this review illustrated a wide range of clinical applications associated with the efficient delivery of CRISPR/ Cas9 gene-editing. Moreover, this article emphasizes the role of the CRISPR/Cas9 system in treating Intra Cerebral haemorrhage (ICH), thereby suggesting future researchers to adopt more clinical trials on this breakthrough delivery system.
CRISPR/Cas-9介导的基因组编辑最近已成为治疗开发和生物医学研究中一项具有潜力和创新性的技术。最近进行了几项研究，以了解获得有效体外结果的基因修饰技术。一般来说，用于基因校正的疾病靶点将位于特定器官，因此了解基因组治疗方法的全部潜力至关重要。从这个角度来看，本综述揭示了CRISPR/Cas9递送系统的重要意义。本综述讨论了两种有前景的基因编辑递送系统，即合成（非病毒）和病毒载体系统。此外，本文试图总结组织特异性和器官特异性mRNA递送系统，为未来的研究人员提供可能的研究信息。此外，还阐明了CRISPR/Cas9系统的主要挑战，如脱靶递送、免疫原性和包装限制。因此，本综述阐述了与CRISPR/Cas9基因编辑有效递送相关的广泛临床应用。此外，本文强调了CRISPR/Cas9系统在治疗脑出血（ICH）中的作用，从而建议未来的研究人员对这一突破性递送系统进行更多的临床试验。
