Gene Therapy Breakthroughs and Challenges

This chapter explores the landscape of gene therapy circa 2004, highlighting both its promising advancements, such as the successful ocular gene therapy LUXTERNA, and the hurdles faced in taking similar therapies to market. The discussion delves into the complexities of regulatory requirements and the impact of corporate decisions on research progress, alongside personal reflections from the speakers on their journey in the field. With insights on adeno-associated viruses and the nuances of gene delivery, the chapter emphasizes the balance between scientific innovation and practical application in treating retinal diseases.