Animal models and different therapies for treatment of retinitis pigmentosa.
Retinitis pigmentosa (RP) is a heterogeneous group of retinal degenerative diseases initially affecting the rod photoreceptor. Patients present with night blindness, loss of peripheral vision and finally the loss of central vision, as a consequence of death of cone photoreceptors. RP is a genetic disease, showing inheritance of autosomal dominant (AD), autosomal recessive (AR) or X-linked (XL) recessive traits, although some patients have no family history of RP (simplex RP). Many animal models of RP are available and have led to a better understanding of the pathology of the disease, and to the development of therapeutic strategies aimed at curing or slowing down the genetic disorder. In this review, we describe the selected animal models (natural and transgenic) and their phenotypes and genotypes, as well as the advantages and disadvantages of the use of each animal. Also, we look at different therapeutic strategies being studied worldwide and report the latest results. Nevertheless, many obstacles will have to be overcome before most of these strategies can be applied to humans.
Rivas
Miren Agurtzane
MA
Department of Cell Biology and Histology, University of the Basque Country, Vizcaya, Spain.
Vecino
Elena
E
D000818:Animals; D030801:Animals, Genetically Modified; D017690:Cell Transplantation; D005808:Genes, Recessive; D015316:Genetic Therapy; D023421:Models, Animal; D020419:Photoreceptor Cells, Vertebrate / Q000473:pathology / Q000637:transplantation; D012174:Retinitis Pigmentosa / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D014815:Vitamins / Q000627:therapeutic use
D014815:Vitamins