title: Human embryonic stem cell-derived cells rescue visual function in dystrophic RCS rats.
abstract: Embryonic stem cells promise to provide a well-characterized and reproducible source of replacement tissue for human clinical studies. An early potential application of this technology is the use of retinal pigment epithelium (RPE) for the treatment of retinal degenerative diseases such as macular degeneration. Here we show the reproducible generation of RPE (67 passageable cultures established from 18 different hES cell lines); batches of RPE derived from NIH-approved hES cells (H9) were tested and shown capable of extensive photoreceptor rescue in an animal model of retinal disease, the Royal College of Surgeons (RCS) rat, in which photoreceptor loss is caused by a defect in the adjacent retinal pigment epithelium. Improvement in visual performance was 100% over untreated controls (spatial acuity was approximately 70% that of normal nondystrophic rats) without evidence of untoward pathology. The use of somatic cell nuclear transfer (SCNT) and/or the creation of banks of reduced complexity human leucocyte antigen (HLA) hES-RPE lines could minimize or eliminate the need for immunosuppressive drugs and/or immunomodulatory protocols.
authors.affiliation: Moran Eye Center, University of Utah Health Science Center, Salt Lake City, Utah, USA.
authors.forename: Raymond D
authors.initials: RD
authors.lastname: Lund
authors.forename: Shaomei
authors.initials: S
authors.lastname: Wang
authors.forename: Irina
authors.initials: I
authors.lastname: Klimanskaya
authors.forename: Toby
authors.initials: T
authors.lastname: Holmes
authors.forename: Rebeca
authors.initials: R
authors.lastname: Ramos-Kelsey
authors.forename: Bin
authors.initials: B
authors.lastname: Lu
authors.forename: Sergej
authors.initials: S
authors.lastname: Girman
authors.forename: N
authors.initials: N
authors.lastname: Bischoff
authors.forename: Yves
authors.initials: Y
authors.lastname: Sauvé
authors.forename: Robert
authors.initials: R
authors.lastname: Lanza
mesh_terms: D000818:Animals; D001483:Base Sequence; D002460:Cell Line; D017931:DNA Primers / Q000235:genetics; D006801:Humans; D010857:Pigment Epithelium of Eye / Q000166:cytology / Q000196:embryology; D051381:Rats; D011922:Rats, Mutant Strains; D012162:Retinal Degeneration / Q000235:genetics / Q000473:pathology / Q000503:physiopathology / Q000628:therapy*; D033581:Stem Cell Transplantation; D013234:Stem Cells / Q000166:cytology* / Q000502:physiology*; D014183:Transplantation, Heterologous
chemical_list: D017931:DNA Primers