This paper from 2021 discusses advancements in gene therapy for inherited retinal diseases. There is potential for the use of adeno-associated viruses (AAVs) for delivering therapeutic genes to retinal cells. However, the authors acknowledge challenges, such as ensuring any gene therapy is delivered to retinal cells effectively. They also note concerns about the long-term safety and stability of potential gene therapy treatments, and possible immune reactions from receiving gene therapies delivered in a viral capsule. The authors explore the significant progress in gene therapy for Usher syndrome in lab-based studies and early clinical trials, showing some promising results.
What this means for Usher syndrome: Gene therapy could be a possible future treatment to address RP in Usher syndrome. However, researchers and scientists still face many hurdles to reach viable treatments. Natural history studies and advances in the field of genetic testing can provide some of the answers needed to expand the number of inherited retinal diseases that can be treated with gene therapies.
