Applications and Research Advances in the Delivery of CRISPR/Cas9 Systems for the Treatment of Inherited Diseases

• DOI: 10.20944/preprints202307.0504.v1
• Publication Date: 2023-07-10T00:56:57Z
• Authors: Xinyue Lu; Miaomiao Zhang; Ge Li; Jingbo Zhang; Xiaoge Fu; Fengying Sun

The rapid advancements in gene therapy have opened up new possibilities for the treatment of genetic disorders, including Duchenne muscular dystrophy, Thalassemia, cystic fibrosis, Hemophilia, and Familial hypercholesterolemia. utilization clustered regularly interspaced short palindromic repeats (CRISPR) - CRISPR-associated protein (Cas) system has revolutionized field by enabling precise targeting genes. In recent years, CRISPR/Cas9 demonstrated remarkable efficacy treating cancer diseases. However, susceptibility nucleic acid drugs to degradation endonucleases, necessitates development functional vectors capable protecting acids from enzymatic degradation, while ensuring safety effectiveness. This review aims explore biomedical potential non-viral vector-explore systems Furthermore, it provides a comprehensive overview research advances viral vector-based insights preclinical clinical studies. Additionally, analyzes current limitations these delivery proposes avenues novel nano-delivery platforms.