Challenges and Opportunities in the Development of Gene Therapies

Abstract

Gene therapy holds immense promise as a revolutionary approach to treating a wide array of genetic disorders, cancers, and infectious diseases by targeting the underlying genetic defects. However, its translation from bench to bedside has been met with numerous challenges. This abstract provides an overview of the major challenges and opportunities in the development of gene therapies. One of the primary challenges is the delivery of therapeutic genes to the target cells with high efficiency and specificity. Various viral and non-viral vectors have been developed, each with its advantages and limitations. Additionally, ensuring the long-term expression of the therapeutic gene without triggering immune responses or insertional mutagenesis remains a significant hurdle