Gene therapy development for retinal diseases.

Gene therapy is being investigated as a potential treatment for a range of inherited and acquired retinal diseases. This chapter provides an overview of current gene therapy strategies, including gene augmentation and gene editing approaches. Techniques targeting both DNA and RNA are discussed, along with considerations for the use of viral and nonviral delivery systems. Challenges such as accurate gene identification, vector optimization, immune responses, and the availability of representative disease models are discussed in detail. Clinical applications are explored in the context of inherited retinal dystrophies, including recent trial outcomes and morphologic and functional endpoints. Overall, the chapter outlines the current state of research and development in retinal gene therapy and summarizes key considerations for clinical translation.