The Use Of Gene Therapy In Pharmaceutical Interventions

Abstract

There has been a renewed interest and increased investment in the field of gene therapy, primarily due to advancements in viral vector technology. This has led to the recent approval by the United States Food and Drug Administration of the first gene therapy product that specifically targets an illness caused by mutations in a single gene. LUXTURNA™ is a therapy that introduces a functional version of the RPE65 gene into retinal cells. It is used to treat retinal dystrophy associated with biallelic RPE65 mutation, a condition that causes blindness. Due to a better knowledge of the genetic causes of ocular disorders and the specific characteristics of the ocular area that make it suitable for local gene therapy, several new gene therapy initiatives are being developed. These programs aim to address hereditary retinal diseases as well as other ocular diseases. In this review, we examine the increasing amount of literature that discusses the creation and advancement of ocular gene therapy products. We focus specifically on the selection of targets and vectors, as well as the chemistry, production, and regulations involved in their development.