Anata Bezman

How can advancements in gene editing techniques revolutionize the future of medicine by enabling new treatments and overcoming curent challenges?

Started July 30, 2024

Abstract or project description

Gene therapy is a new modality of medical treatment that can treat or prevent diseases by directly modifying gene expression. Among the various approaches to gene therapy, four mechanisms that stand out for their therapeutic benefits include small interfering RNAs (siRNAs), small activating RNAs (saRNAs), micro RNAs (miRNAs), and the CRISPR/Cas system. Both siRNA and miRNA function as gene expression silencers, as siRNA targets specific mRNA for degradation and miRNA represses protein production. On the other hand, saRNA promotes gene expression by targeting the promoter regions and increasing transcription rates. The CRISPR/Cas system edits the gene by inserting, deleting, or correcting specific sequences. These mechanisms provide different approaches for gene silencing, correction, and activation while offering therapeutic potential in many disorders directly impacted by genetics. However, gene therapy is a largely debated approach due to challenges in the mechanisms and viabilities of the many approaches. This review paper will provide a comprehensive evaluation of four innovative gene therapy approaches via their mechanisms of action, advantages, and challenges, as well as some notable applications for the treatment of various diseases.