title: Eyes open to stem cells: safety trial may pave the way for cell therapy to treat retinal disease in patients.
abstract: A clinical trial using human embryonic stem cell (hESC) therapy for an inherited retinal degenerative disease is about to commence. The Advanced Cell Technology (ACT) trial will treat patients with Stargardt's macular dystrophy using transplanted retinal pigment epithelium derived from hESCs. Currently, no effective treatment is available for Stargardt's disease so a stem cell-based therapy that can slow progression of this blinding condition could represent a significant breakthrough. While there are some hurdles to clear, the ACT trial is a fine example of translational research that could eventually pave the way for a range of stem cell therapies for the retina and other tissues.
authors.forename: Reinhold J
authors.initials: RJ
authors.lastname: Medina
authors.forename: Desmond B
authors.initials: DB
authors.lastname: Archer
authors.forename: Alan W
authors.initials: AW
authors.lastname: Stitt
mesh_terms: D018528:ATP-Binding Cassette Transporters / Q000235:genetics / Q000378:metabolism; D002454:Cell Differentiation; D064987:Cell- and Tissue-Based Therapy; D002986:Clinical Trials as Topic; D003317:Corneal Dystrophies, Hereditary / Q000235:genetics / Q000628:therapy*; D053595:Embryonic Stem Cells / Q000166:cytology / Q000637:transplantation; D006801:Humans; D009154:Mutation; D055213:Retinal Pigment Epithelium / Q000166:cytology
chemical_list: C056475:ABCA4 protein, human; D018528:ATP-Binding Cassette Transporters