Effects of Adeno-associated Virus-vectored Ciliary Neurotrophic Factor on Retinal Structure and Function in Mice with a P216L rds/peripherin Mutation

• Published in: Experimental eye research Vol. 74; no. 6; pp. 719 - 735
• Main Authors: Bok, Dean, Yasumura, Douglas, Matthes, Michael T., Ruiz, Alberto, Duncan, Jacque L., Chappelow, Aimee V., Zolutukhin, Sergei, Hauswirth, William, LaVail, Matthew M.
• Format: Journal Article
• Language: English
• Published: London Elsevier Ltd 01.06.2002; Elsevier
• Subjects: AAV; Adenoviridae - genetics; Animals; Biological and medical sciences; ciliary neurotrophic factor; Ciliary Neurotrophic Factor - genetics; Ciliary Neurotrophic Factor - physiology; CNTF; cytokine; Electroretinography; Eye and associated structures. Visual pathways and centers. Vision; Eye Proteins - genetics; Fundamental and applied biological sciences. Psychology; gene therapy; Genetic Therapy - methods; Genetic Vectors; Intermediate Filament Proteins - genetics; Membrane Glycoproteins; Mice; Mice, Inbred C57BL; Mice, Transgenic; Nerve Tissue Proteins - genetics; Peripherins; photoreceptor; Photoreceptor Cells, Vertebrate - pathology; Point Mutation; rds; rescue; Retina - physiopathology; Retinal Degeneration - pathology; Retinal Degeneration - physiopathology; Retinal Degeneration - therapy; retinitis pigmentosa; Reverse Transcriptase Polymerase Chain Reaction; Vertebrates: nervous system and sense organs; rds; cytokine; CNTF; AAV; photoreceptor; retinitis pigmentosa; gene therapy; mice; ciliary neurotrophic factor; rescue; Eye; Visual system; Neurotrophic factor; Vertebrata; Mammalia; Mouse; Rodentia; Peripherin; Retina; Mutation; Ciliary neurotrophic factor

Past studies have shown that acute administration of ciliary neurotrophic factor (CNTF) can prolong the survival of retinal photoreceptor cells that have undergone phototoxic injury or that express gene mutations. Adenovirus-vectored CNTF has also been effective but for all of these treatments, the...