Gene Editing: A New Approach to Treat Genetic Disorders
DOI:
https://doi.org/10.58445/rars.449Keywords:
Gene Editing, CRISPR, Genetic Disorders, Sickle Cell Disease, Mutations, Genomic Medicine, Preclinical studiesAbstract
Gene editing has emerged as a groundbreaking method in tackling genetic disorders/diseases by precisely altering DNA sequences. Genetic disorders like Turner Syndrome and Sickle cell disease occur from mutations or altered genetic material. Gene editing technologies, including TALENs, ZFNs, and CRISPR-Cas9, have introduced the power to fix mutations at precise locations in the genome. The CRISPR-Cas9 system can efficiently modify genes with unprecedented precision. As shown by research conducted on Leber Congenital Amaurosis type 10 and Sickle cell disease, gene editing holds promise for correcting disease-causing mutations. However, ethical concerns and safety considerations, including germline editing and off-target effects, indicate that caution is necessary. As the field moves forward, recent advancements like prime editing and tackling antimicrobial resistance highlight the hidden potential of gene editing. Gene editing will continue to grow and unleash a field of innovative and personalized medicine, revolutionizing healthcare.
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