Personalized Gene Therapy

Images of normal (above) and diseased retinas. Patients with MFRP mutations, a cause of retinitis pigmentosa, lose the function of most retinal cells, particularly at the periphery of the retina, leaving them with drastically reduced vision. Personalized gene therapy, using iPS cells, may offer a way to correct this genetic disorder.
Patient-Specific Stem Cells and Personalized Gene Therapy