Abstract
Human genetic disorders, arising from a range of genetic irregularities, can significantly affect human physiology, often with limited available treatment options. The development of the CRISPR system, facilitating precise editing of the genome, has opened new avenues for addressing a range of mutations found in various genetic disorders. However, there is currently a lack of comprehensive reviews that specifically address the application of CRISPR in genetic diseases. To bridge this gap, this review focuses on exploring the advancements in CRISPR technology and their utility in therapeutic approaches for various genetic disorders. This review introduces human genetic disorders, explains the fundamental mechanisms of CRISPR editing, and highlights the latest advancements in CRISPR technology. Additionally, it examines three CRISPR delivery techniques, including physical delivery, viral vectors, and nanocarriers. It further reviews CRISPR's applications in therapeutic approaches for genetic disorders. Finally, it identifies the primary hurdles associated with industrial development and ethics considerations that should be addressed before the application of CRISPR in a medical context.
| Original language | English |
|---|---|
| Article number | 2402630 |
| Pages (from-to) | 1-19 |
| Number of pages | 19 |
| Journal | Advanced Functional Materials |
| Volume | 34 |
| Issue number | 38 |
| Early online date | 27 May 2024 |
| DOIs | |
| Publication status | Published - Sept 2024 |
| Externally published | Yes |
Bibliographical note
Copyright the Author(s) 2024. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher.Keywords
- CRISPR gene editing
- gene delivery system
- gene therapy
- genetic diseases