Application of CRISPR gene editing technology in cystic fibrosis treatment

Abstract

CRISPR-Cas9 gene editing technology is to identify the target genome sequence through artificially designed sgRNA, guide Cas9 protease, so that it can effectively cut the DNA double strand, form a double strand break, repair after damage to lead to gene knockout or knock-in, and finally achieve the purpose of modifying genomic DNA which leading CRISPR a technology can effectively repair, correct or regulate genetic mutations associated with genetic diseases, providing patients with new treatment options. However, despite the remarkable progress, CRISPR technology still faces many challenges in its clinical application, including ethics and safety. This article reviews the principle and development of CRISPR technology, as well as its application in the treatment of cystic fibrosis, and summarizes the challenges of CRISPR in clinical applications. More importantly, this article focuses on some current improvements to CRIPSR to make CRISPR gene editing technology more valuable and meaningful in the treatment of genetic diseases in the future.