Paper Key : IRJ************766
Author: Shubham Kailas Ingole
Date Published: 22 Nov 2024
Abstract
Gene therapy has emerged as a promising approach to treating rare diseases, many of which are genetic and currently lack effective therapeutic options. This review explores the mechanisms, advances, and challenges associated with gene therapy in rare diseases, focusing on strategies like in vivo and ex vivo approaches, gene editing, and RNA interference. Notable successes in rare conditionssuch as spinal muscular atrophy (SMA), hemophilia, and Lebers congenital amaurosishighlight the clinical potential of gene therapies. Delivery systems, primarily using viral vectors like adeno-associated viruses (AAV), have proven effective, though immune responses and tissue-specific targeting remain obstacles. Challenges include high development costs, regulatory hurdles, and ethical considerations, particularly in gene editing applications. Innovations in vector engineering, CRISPR-based gene editing, and enhanced delivery methods indicate a promising future, potentially expanding treatment access to more rare diseases. Overall, gene therapy continues to demonstrate transformative potential, but achieving widespread access and ensuring safety will be key for its successful integration into clinical practice