CRISPR/Cas system in human genetic diseases

Liuyijia Zhang

doi:10.1117/12.3012830

8 January 2024 CRISPR/Cas system in human genetic diseases

Liuyijia Zhang

Author Affiliations +

Proceedings Volume 12924, Third International Conference on Biological Engineering and Medical Science (ICBioMed2023); 129241B (2024) https://doi.org/10.1117/12.3012830
Event: 3rd International Conference on Biological Engineering and Medical Science (ICBioMed2023), 2023, ONLINE, United Kingdom

Abstract

Diseases caused by genetic disorder are in major concern of the world and the situation is even more severe as aging. Scientists are seeking for approaches to prevent and treat various genetic diseases. For therapeutic purposes, gene therapy is used as a gene editing technology to "modify" cell genes, and recent advances in understanding of gene therapy offer some solutions. CRISPR/Cas system, compared to the previous two generations of gene-editing technologies, provides a straightforward, efficient and adaptable platform. At the same time, novel CRISPR/Cas technologies, including Cas13 effectors, base editors, and prime editors, have been developed to counteract the negative effects of double-stranded DNA breaks. Currently, CRISPR/Cas system and its derivatives are widely employed in the synthesis of animal models, drug target searching, and gene function investigation, and they also offer various potential applications in gene therapy. Although efficiency and specificity with which the CRISPR/Cas system is develivered into target sequences need to be improved, it offers a strong tool for gene editing and therefore treatment of genetic disease. In this review, not only the classification, molecular mechanisms involved in different CRISPR/Cas systems were systematically introduced, but also the the application of the system in various genetic diseases was discussed.

Citation Download Citation

Liuyijia Zhang "CRISPR/Cas system in human genetic diseases", Proc. SPIE 12924, Third International Conference on Biological Engineering and Medical Science (ICBioMed2023), 129241B (8 January 2024); https://doi.org/10.1117/12.3012830

ACCESS THE FULL ARTICLE

INSTITUTIONAL
Select your institution to access the SPIE Digital Library.

SELECT YOUR INSTITUTION

PERSONAL
Sign in with your SPIE account to access your personal subscriptions or to use specific features such as save to my library, sign up for alerts, save searches, etc.

PERSONAL SIGN IN

No SPIE Account? Create one

;

PURCHASE THIS CONTENT

SUBSCRIBE TO DIGITAL LIBRARY

50 downloads per 1-year subscription

Members: $195

Non-members: $335 ADD TO CART

25 downloads per 1 - year subscription

Members: $145

Non-members: $250 ADD TO CART

PURCHASE SINGLE ARTICLE