Biotechnological Approaches to Rare Genetic Disorders: Innovations and Challenges

Abstract

Biotechnological approaches have significantly advanced the understanding and treatment of rare genetic disorders. These conditions, often characterized by their low prevalence and genetic underpinnings, pose unique challenges that require innovative solutions. Recent developments in gene therapy, genome editing, and personalized medicine have shown promising results in managing and potentially curing these disorders. This article reviews current biotechnological strategies, explores the associated challenges, and discusses future directions for research and application. Emphasis is placed on cutting-edge techniques, including CRISPR/Cas9, viral vector-based therapies, and innovative drug delivery systems. The article also addresses ethical, regulatory, and logistical considerations critical to advancing these therapies from the laboratory to clinical practice.