J Electrodiagn Neuromuscul Dis.
2025 Apr;27(1):1-5. 10.18214/jend.2024.00073.
Therapeutic Applications of Genome Editing Tools for Genetic Diseases
- Affiliations
-
- 1Genomic Medicine Institute, Seoul National University College of Medicine, Seoul, Korea
- 2Department of Biomedical Sciences, Seoul National University College of Medicine, Seoul, Korea
- 3Cancer Research Institute, Seoul National University College of Medicine, Seoul, Korea
- 4Institute of Molecular Biology and Genetics, Seoul National University, Seoul, Korea
- KMID: 2567486
- DOI: http://doi.org/10.18214/jend.2024.00073
Abstract
- Since the discovery of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated (Cas) genome editing technologies, significant efforts have been made to improve their efficiency and accuracy. Consequently, advanced tools such as base editors (BEs) and prime editors (PEs) have emerged over the past decade. Today, CRISPR, BE, and PE systems are actively employed in treating genetic diseases. Notably, CASGEVY by CRISPR therapeutics—the world’s first gene editing treatment using CRISPR technology—received approval from the Food and Drug Administration of both the United Kingdom and the United States last year. This review highlights recent advancements in various CRISPR technologies for therapeutic applications in genetic diseases.
- Full Text Links
-
- Actions
-
Cited
- CITED
-
- Close
- Share
-
- Similar articles
-
- Basic Principles and Clinical Applications of CRISPR-Based Genome Editing
- Applications of CRISPR/Cas9 for Gene Editing in Hereditary Movement Disorders
- Genome editing: the road of CRISPR/Cas9 from bench to clinic
- Recent Research Trends in Stem Cells Using CRISPR/Cas-Based Genome Editing Methods
- Application of CRISPR-Cas9 gene editing for congenital heart disease
