Suppression of rds expression by siRNA and gene replacement strategies for gene therapy using rAAV vector

Advances in Experimental Medicine and Biology

Volumn 723, Issue , 2012, Pages 215-223

  Petrs Silva, Hilda ^a   Yasumura, Douglas ^b   Matthes, Michael T ^b   LaVail, Matthew M ^b   Lewin, Alfred S ^a,c   Hauswirth, William W ^a  

^a FEDERAL UNIVERSITY OF RIO DE JANEIRO   (Brazil)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c UNIVERSITY OF FLORIDA   (United States)

Author keywords

Adeno Associated Viral Vector; Gene Replacement; Gene Therapy; Photoreceptor Degeneration; rds Peripherin; Retinitis Pigmentosa; siRNA

Indexed keywords

MESSENGER RNA; PARVOVIRUS VECTOR; PERIPHERIN; SMALL INTERFERING RNA;

ANIMAL EXPERIMENT; ANIMAL MODEL; AUTOSOMAL DOMINANT DISORDER; AUTOSOMAL DOMINANT RETINITIS PIGMENTOSA; CELL STRAIN HEK293; CONFERENCE PAPER; CONTROLLED STUDY; GENE; GENE EXPRESSION; GENE MUTATION; GENE REPLACEMENT THERAPY; GENETIC TRANSDUCTION; GENETIC TRANSFECTION; IN VITRO STUDY; IN VIVO STUDY; MOUSE; NONHUMAN; PHOTORECEPTOR CELL; PRIORITY JOURNAL; RDS GENE; REAL TIME POLYMERASE CHAIN REACTION; RETINITIS PIGMENTOSA; RNA ISOLATION; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; ELECTRORETINOGRAPHY; GENE THERAPY; GENES, DOMINANT; HEK293 CELLS; HUMANS; INTERMEDIATE FILAMENT PROTEINS; MEMBRANE GLYCOPROTEINS; MICE; NERVE TISSUE PROTEINS; PHOTORECEPTOR CELLS, VERTEBRATE; RETINITIS PIGMENTOSA; RNA, MESSENGER; RNA, SMALL INTERFERING;

ADENO-ASSOCIATED VIRUS; MUS;

EID: 84855931333     PISSN: 00652598     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-4614-0631-0_29     Document Type: Conference Paper

References (10)

1. Ali RR, Sarra GM, Stephens C et al (2000) Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. Nat Genet 25:306-310 (Pubitemid 30437318)
2. Fingert JH, Oh K, Chung M et al (2008) Association of a novel mutation in the retinol dehydrogenase 12 (RDH12) gene with autosomal dominant retinitis pigmentosa. Arch Ophthalmol 126: 1301-1307
3. Hammond SM, Bernstein E, Beach D, Hannon GJ (2000) An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophila cells. Nature 404:293-296 (Pubitemid 30163547)
4. Hartong DT, Berson EL, Dryja TP (2006) Retinitis pigmentosa. Lancet 368:1795-809 (Pubitemid 44739028)
5. Ma J, Norton JC, Allen AC et al (1995) Retinal degeneration slow (rds) in mouse results from simple insertion of a t haplotype-specifi c element into protein-coding exon II. Genomics 28:212-219
6. Sarra GM, Stephens C, de Alwis M et al (2001) Gene replacement therapy in the retinal degeneration slow (rds) mouse: the effect on retinal degeneration following partial transduction of the retina. Hum Mol Genet 10:2353-2361 (Pubitemid 33069541)
7. Schlichtenbrede FC, da Cruz L, Stephens C et al (2003) Long-term evaluation of retinal function in Prph2Rd2/Rd2 mice following AAV-mediated gene replacement therapy. J Gene Med 5: 757-764 (Pubitemid 40309713)
8. Timmers AM, Zhang H, Squitieri A et al (2001) Subretinal injections in rodent eyes: effects on electrophysiology and histology of rat retina. Mol Vis 7:131-137 (Pubitemid 36153662)
9. Wrigley JD, Ahmed T, Nevett CL et al (2000) Peripherin/rds infl uences membrane vesicle morphology. Implications for retinopathies. J Biol Chem 275:13191-13194 (Pubitemid 30257372)
10. Zolotukhin S, Potter M, Zolotukhin I et al (2002) Production and purifi cation of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods 28:158-167 (Pubitemid 35341079)