Gene therapy for retinal degeneration.
Inherited retinal degenerations are a group of diseases leading to blindness through progressive loss of vision in many patients. Although with the cloning of more and more disease genes the knowledge on the molecular genetics of these conditions and on the apoptotic pathway as the common disease mechanism is steadily increasing, there is still no cure for those affected. In recent years, new experimental treatments have evolved through the efforts of many investigators and have been explored in animal models. The rationale of the different strategies for developing a treatment based on gene replacement or rescue of the diseased neuronal tissue with growth factors will be outlined and discussed in this paper.
Reichel
M B
MB
Department of Ophthalmology, Faculty of Medicine, University of Leipzig, Deutschland.
Ali
R R
RR
Hunt
D M
DM
Bhattacharya
S S
SS
D000256:Adenoviridae / Q000235:genetics; D000818:Animals; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D007381:Intermediate Filament Proteins / Q000235:genetics; D008562:Membrane Glycoproteins; D009419:Nerve Tissue Proteins / Q000235:genetics; D064531:Peripherins; D012160:Retina / Q000473:pathology / Q000821:virology; D012162:Retinal Degeneration / Q000235:genetics / Q000473:pathology / Q000628:therapy*
D007381:Intermediate Filament Proteins; D008562:Membrane Glycoproteins; D009419:Nerve Tissue Proteins; D064531:Peripherins