Affiliation:
1. School of Biomedical Engineering University of Technology Sydney Ultimo New South Wales 2007 Australia
2. Graduate School of Biomedical Engineering ARC Centre of Excellence in Nanoscale Biophotonics Faculty of Engineering UNSW Sydney Sydney New South Wales 2052 Australia
3. Ingham Institute for Applied Medical Research Liverpool New South Wales 2170 Australia
4. Cancer Diagnosis and Pathology Group Kolling Institute of Medical Research Royal North Shore Hospital St Leonards Sydney New South Wales 2065 Australia
5. Sydney Medical School University of Sydney Sydney New South Wales 2006 Australia
Abstract
AbstractHuman 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.