CRISPR-Cas9 Treatment for People with Genetic Disorders
DOI:
https://doi.org/10.58445/rars.3453Keywords:
CRISPR/Cas9, Genetic disorders, Gene editingAbstract
CRISPR/Cas 9 is a new gene editing technology that can make precise changes in DNA. The CRISPR method is based on a natural system used by bacteria to protect themselves from viruses. It has shown great potential in treating genetic disorders: Down syndrome, by targeting extra copies of chromosome 21; sickle cell disease, by correcting mutations in the beta-globin gene to restore fetal hemoglobin; and CPS1 (Carbamoyl Phosphate Synthetase 1) deficiency, by fixing mutations that cause toxic accumulation of ammonia in the body. While CRISPR is a promising tool, the technology is still considered relatively new and can be further developed. This review aims to analyze the potential applications of CRISPR from the cellular level to real-life medical treatment, as well as the concerns of its usage. The paper also discusses each application of CRISPR in a range of genetic disorders. Understanding these aspects is crucial for future research and ensuring responsible implementations of CRISPR in medicine. One recent CRISPR advancement, including the first FDA-approved CRISPR-based therapy(Casgevy) and eventually, there will be further development using CRISPR.
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