Due to the advantages of simple design, low cost, high efficiency, good repeatability and short-cycle, CRISPR-Cas systems have become the most widely used genome editing technology in molecular biology laboratories all around the world. In this review, an overview of the CRISPR-Cas systems will be introduced, including the innovations, the applications in human disease research and gene therapy, as well as the challenges and opportunities that will be faced in the practical application of CRISPR-Cas systems.
Double-strand break (DSB) induced by Link Slot Gacor Mudah Menang nucleases can be repaired by non-homologous end joining (NHEJ) or homology-directed repair (HDR) pathways. NHEJ can introduce random insertions or deletions (indels) of varying length at the site of the DSB.
CRISPR/Cas9 can make a single cut using one guide RNA; the cut is then repaired through natural processes, which can result in the addition or deletion of base pairs, leading to gene inactivation.
Many diseases, both rare and common, have a genetic basis. The scientific understanding of how specific genes are involved in disease is advancing rapidly, offering the opportunity to use gene editing technologies to disrupt or correct disease-related genes.
In biomedical research, CRISPR-Cas9 has facilitated the study of gene functions and disease mechanisms. It has enabled researchers to create targeted gene knockouts, generate disease models, and explore potential therapeutic strategies.
For instance, in a study by Hsu et al., CRISPR-Cas9 was used to successfully edit multiplegenes simultaneously, providing a powerful tool for functional genomics research[7].CRISPR-Cas9 has also found applications in agriculture, bioinformatics, and biotechnology. In agriculture, it offers a promising approach for crop improvement by modifying genes related to disease resistance, yield, and nutritional content.