Recent advances in CRISPR/Cas9-assisted gene therapy

Abstract

CRISPR/Cas9 (Clustered regularly interspaced short palindromic repeats) is an exponentially growing tool with wide-spread applications in therapeutics like gene modifications that focus on altering the hereditary material to repair or eliminate any defective gene-causing diseases like cancer, AIDS (Acquired immunodeficiency syndrome), etc. It also includes the identification of the target sequence with the help of sgRNA followed by the substitution of a malfunction-ing gene with a normal version. It offers high efficiency, specificity, and post-gene-editing efficacy, but have also some off-target impressions, and immunogenic effects. The contribution of CRISPR/Cas9 has already been proved primarily in in-vitro studies using animal germ cell lines but translation in in-vivo models is still not much supported due to ethi-cal considerations. The recent advances include studies and clinical trials focusing on the treatment of various diseases of genetic origin. For instance, CRISPR gene knock-in technique was applied for in-vivo Leber Congenital Amaurosis 10 treatment, where CRISPR components were delivered via sub-retinal injection to correct the mutation in CE9290. The current paper recapitulates the capability of CRISPR/Cas9 in in-vivo gene therapy for various disorders like cancer, AIDS, sickle cell disease and the most recent COVID-19. The insights presented herein are poised to contribute signifi-cantly to the advancement of the field, fostering a deeper understanding of CRISPR/Cas9 technology and accelerating its clinical transition. Ultimately, this review paper serves as a valuable resource for researchers, clinicians, and policy-makers invested in the continued evolution of gene therapy and responsible utilization of CRISPR/Cas9 for human welfare