Challenges of Treatment Methodologies and the Future of Gene Therapy and Stem Cell Therapy to Treat Retinitis Pigmentosa.

Retinitis pigmentosa (RP) is a heterogeneous group of hereditary retinal degenerations for which there is currently no cure. Studies investigating the use of gene therapy, gene editing, and stem cells as potential treatment strategies have shown promising results in animal models and some early clinical trials. Even still, major barriers still exist, including the ability to develop therapies that can target the wide range of mutational etiologies and phenotypic presentations that encompass RP. Additionally, effective screening and early diagnosis are crucial for maximum therapeutic potential, especially because many therapeutic agents require a baseline level photoreceptor function.