CRISPR-CAS9 APPLICATIONS IN TREATING MONOGENIC DISORDERS: PROMISE AND PERILS

Authors

  • Dr. Samina Khalid Department of Genetics, Quaid-i-Azam University, Islamabad, Pakistan. Author

DOI:

https://doi.org/10.71465/jhmri33

Keywords:

CRISPR-Cas9, Monogenic Disorders, Gene Editing, Genetic Therapy

Abstract

CRISPR-Cas9 technology has emerged as a revolutionary gene-editing platform with vast implications in precision medicine. This review examines its application in treating monogenic disorders such as cystic fibrosis, sickle cell anemia, and Duchenne muscular dystrophy. The paper explores the underlying mechanism of CRISPR-Cas9, recent breakthroughs in clinical trials, delivery systems, and ethical issues associated with germline editing. While significant therapeutic success has been documented, technical challenges like off-target effects and immunogenic responses warrant cautious advancement. This paper provides a comprehensive overview of both the potential and limitations of CRISPR-Cas9 for curing monogenic diseases.

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Published

2024-09-30

Issue

Vol. 2 No. 3 (2024): Journal of Health, Medical Research and Innovations

Section

Articles

License

Creative Commons License

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