Impact of Gene Therapy and Genetic Mutations in Rare Diseases

Abstract

This article provides a thorough examination of recent progress in gene therapy as a transformative avenue for treating rare diseases characterized by genetic mutations. The authors explore fundamental principles of gene therapy, encompassing both viral and non-viral vectors employed for efficient gene delivery. This article delves into the potential of state-of-the-art genome-editing technologies, such as CRISPR/Cas9, to rectify genetic abnormalities, emphasizing their precision and scalability. Regulatory considerations and ethical implications surrounding gene therapy are discussed, underlining the importance of a balanced and responsible approach in translating these innovations into clinical practice. As gene therapy continues to evolve, its expanding role in addressing rare diseases offers hope to individuals grappling with these complex conditions.