Advancements in Gene Therapy for Rare Genetic Disorders

Abstract

Thanks to the tremendous advancements in gene therapy in the past few years, patients with rare genetic diseases now have a new reason to hope. gives a brief synopsis of current advances in gene therapy for different diseases. Rare hereditary disorders, which impact a tiny fraction of the population but render individuals extremely vulnerable, are typically caused by errors in a single gene. Prior to recently, doctors could only alleviate the symptoms of these conditions; they couldn't fix the underlying genetic defects that brought them on. However, a new era of precision medicine had begun with the advent of gene therapy, which would forever change the approach to treating uncommon diseases. Recent advances in gene therapy have showed hope as a means of treating or alleviating uncommon ailments resulting from genetic abnormalities. These innovations have been developed using state-of-the-art methods such as viral vectors, RNA-based treatments, and CRISPR-Cas9 gene editing. Trials have shown that these techniques have the potential to be therapeutically useful. Participants in these studies came from all walks of life and suffered from uncommon genetic illnesses.