Gene Therapy Using Stem Cells

Abstract

Viral-mediated gene augmentation therapy has recently shown success in restoring vision to patients with retinal degenerative disorders. Key to this success was the availability of animal models that accurately presented the human phenotype to test preclinical efficacy and safety. These exciting studies support the use of gene therapy in the treatment of devastating retinal degenerative diseases. In some cases, however, in vivo gene therapy for retinal degeneration would not be effective because the cell types targeted are no longer present. The development of somatic cell reprogramming methods provides an attractive source of autologous cells for transplantation and treatment of retinal degenerative disease. This article explores the development of gene therapy and patient-derived stem cells for the purpose of restoring vision to individuals suffering from inherited retinal degenerations.