CRISPR/Cas system in human genetic diseases
Year: 2024
Type: article
Abstract: The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system is an adaptive immune system derived from bacteria which is designed to recognize and cleave invading DNA, such as viruses and plasmids. The advent of this innovative gene-editing technology has emerged as influential force in the category of medicine, delivering unprecedented precision in targeting and modifying genetic material. This review explores the potential and challenges of CRISPR in the context of human diseases. Specific applications in diseases affecting hemoglobin, muscle, the eye, and the liver are examined, demonstrating the versatility of CRISPR in addressing both monogenic and complex diseases. The use of CRISPR for gene therapy in conditions like sickle cell disease, Duchenne muscular dystrophy, retinal degeneration, and liver disorders is discussed, showcasing early clinical trials and preclinical studies that underscore the promise of CRISPR in medicine. Finally, it is expected that CRISPR will be able to play a greater role in human health and bring more precise and personalized treatment plans to patients.
Source: Theoretical and Natural Science
Author: Haoyue Zhang
Institution: Sun Yat-sen University
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: hybrid
OpenAlex ID: W4403378909