human pigment epithelium-derived factor gene therapy

Human pigment epithelium-derived factor (PEDF) gene therapy is an investigational neuroprotective gene therapy designed to treat retinitis pigmentosa (RP). Developed by the National Hospital Organization Tokyo Medical Center with funding and support from the Japan Agency for Medical Research and Development (AMED), the therapy employs an adeno-associated virus serotype 2 (AAV2) vector to deliver the human SERPINF1 gene directly to the subretinal space. The gene encodes PEDF, a secreted glycoprotein with potent neurotrophic, anti-apoptotic, and anti-oxidative properties. By ensuring continuous production of PEDF within the retina, the therapy aims to preserve the survival and function of photoreceptor cells, thereby delaying the progression of vision loss in patients with RP, regardless of their underlying genetic mutation.

Other names

PEDF gene therapyAAV-PEDF