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Advancements in CRISPR Technologies and Treatment of Genetic Disorders
- 1 University of Toronto
* Author to whom correspondence should be addressed.
Abstract
When CRISPR-Cas9, short for Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) with CRISPR-associated protein 9 (Cas9), was successfully harnessed for genome editing in the early 2010s, it marked a new era for biotechnology. The high precision, efficiency, and adaptability of CRISPR-Cas9 have unlocked extraordinary potential in medicine, agriculture, and industrial biology, underscored by the awarding of the Nobel Prize in Chemistry in 2020 to its pioneers. This paper reviews follow-on advancements to the technology addressing challenges, including off-target effects and inefficient delivery systems, and explores its transformative applications in treating genetic disorders, including sickle cell disease, transfusion-dependent β-thalassemia, and cystic fibrosis. Additionally, it highlights ongoing hurdles management of such as high costs and safety and efficacy of heritable gene editing. This study shows that addressing these challenges and fostering ethical and collaborative advancements will be essential for CRISPR technologies, which can fulfill their transformative potential in improving human life quality.
Keywords
CRISPR-Cas9, genetic disorders, genome editing, biotechnology, medicine
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Cite this article
Chen,Y. (2025). Advancements in CRISPR Technologies and Treatment of Genetic Disorders. Theoretical and Natural Science,96,20-26.
Data availability
The datasets used and/or analyzed during the current study will be available from the authors upon reasonable request.
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Volume title: Proceedings of the 5th International Conference on Biological Engineering and Medical Science
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