Institute of Microbiology University of Agriculture, Faisalabad, Pakistan.
Center of Agricultural Biochemistry and Biotechnology, University of Agriculture, Faisalabad, Pakistan.
J Biomed Sci. 2018 Mar 28;25(1):29. doi: 10.1186/s12929-018-0425-5.
Advances in Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR associated system (CRISPR/Cas9) has dramatically reshaped our ability to edit genomes. The scientific community is using CRISPR/Cas9 for various biotechnological and medical purposes. One of its most important uses is developing potential therapeutic strategies against diseases. CRISPR/Cas9 based approaches have been increasingly applied to the treatment of human diseases like cancer, genetic, immunological and neurological disorders and viral diseases. These strategies using CRISPR/Cas9 are not only therapy oriented but can also be used for disease modeling as well, which in turn can lead to the improved understanding of mechanisms of various infectious and genetic diseases. In addition, CRISPR/Cas9 system can also be used as programmable antibiotics to kill the bacteria sequence specifically and therefore can bypass multidrug resistance. Furthermore, CRISPR/Cas9 based gene drive may also hold the potential to limit the spread of vector borne diseases. This bacterial and archaeal adaptive immune system might be a therapeutic answer to previous incurable diseases, of course rigorous testing is required to corroborate these claims. In this review, we provide an insight about the recent developments using CRISPR/Cas9 against various diseases with respect to disease modeling and treatment, and what future perspectives should be noted while using this technology.
CRISPR 相关系统(CRISPR/Cas9)的进展极大地改变了我们编辑基因组的能力。科学界正在将 CRISPR/Cas9 用于各种生物技术和医学目的。其最重要的用途之一是开发针对疾病的潜在治疗策略。基于 CRISPR/Cas9 的方法已越来越多地应用于治疗癌症、遗传、免疫和神经疾病以及病毒疾病等人类疾病。这些使用 CRISPR/Cas9 的策略不仅针对治疗,还可用于疾病建模,这反过来又可以提高对各种传染病和遗传疾病机制的理解。此外,CRISPR/Cas9 系统还可以用作可编程抗生素,特异性杀死细菌序列,从而可以绕过多药耐药性。此外,基于 CRISPR/Cas9 的基因驱动也可能具有限制媒介传播疾病传播的潜力。这种细菌和古菌适应性免疫系统可能是对以前无法治愈疾病的治疗方法,当然需要进行严格的测试来证实这些说法。在这篇综述中,我们提供了关于使用 CRISPR/Cas9 针对各种疾病进行疾病建模和治疗的最新进展的见解,以及在使用这项技术时应注意的未来前景。