Gene Editing and Its Application in Healthcare

Abstract

Gene editing, particularly through CRISPR-Cas9 technology, represents a transformative advancement in medicine, agriculture, and research. CRISPR-Cas9 works by using a guide RNA (gRNA) to precisely target a specific DNA sequence, allowing the Cas9 enzyme to cut the DNA at that location. The cell then repairs the break, either introducing small mutations or using a provided template for precise corrections. This ability to accurately modify genes has made CRISPR-Cas9 a powerful tool for addressing genetic disorders like cystic fibrosis and sickle cell disease, offering potential cures by correcting the underlying genetic defects.

However, the rapid development of this technology also raises significant safety and ethical concerns. The possibility of off-target effects, where unintended parts of the genome are edited, poses a serious risk, and the prospect of germline editing introduces ethical questions about long-term impacts on future generations. This paper explores CRISPR-Cas9’s potential and the critical considerations for its responsible use.