Long-term effects of retinal gene therapy in childhood blindness

New England Journal of Medicine

Volumn 372, Issue 20, 2015, Pages 1954-1955

  Wright, Alan F ^a  

^a UNIVERSITY OF EDINBURGH   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; PARVOVIRUS VECTOR; PEPTIDES AND PROTEINS; PROTEIN RPE65; UNCLASSIFIED DRUG; CIS TRANS ISOMERASE; RETINOID ISOMEROHYDROLASE;

BLINDNESS; CHILDHOOD BLINDNESS; CHILDHOOD DISEASE; DARK ADAPTATION; DISEASE ASSOCIATION; DOG; DOSE RESPONSE; DRUG ADMINISTRATION ROUTE; DRUG DOSE ESCALATION; DRUG MEGADOSE; EDITORIAL; ELECTRORETINOGRAPHY; EXTRAFOVEAL VISION; FOLLOW UP; GENE; GENE EXPRESSION; GENE THERAPY; HUMAN; IMMUNE RESPONSE; ISOMERIZATION; LEBER CONGENITAL AMAUROSIS; LOSS OF FUNCTION MUTATION; LOW DRUG DOSE; MACULAR THICKNESS; MACULAR THINNING; NONHUMAN; PHOTORECEPTOR; PIGMENT EPITHELIUM; PRIORITY JOURNAL; RETINA; RETINAL GENE THERAPY; ROD SENSITIVITY; RPE65 GENE; TREATMENT DURATION; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM; VIRUS RECOMBINANT; VISION; VISUAL ACUITY; VISUAL SENSITIVITY; VISUAL SYSTEM PARAMETERS; ANIMAL; GENETICS; PATHOLOGY; PHOTORECEPTOR CELL;

ANIMALS; CIS-TRANS-ISOMERASES; DNA, COMPLEMENTARY; GENETIC THERAPY; HUMANS; LEBER CONGENITAL AMAUROSIS; PHOTORECEPTOR CELLS, VERTEBRATE;

EID: 84929353130     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMe1503419     Document Type: Editorial

References (10)

1. Cai X, Conley SM, Naash MI. RPE65: role in the visual cycle, human retinal disease, and gene therapy. Ophthalmic Genet 2009;30:57-62.
2. Redmond TM, Yu S, Lee E, et al. Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle. Nat Genet 1998;20:344-51.
3. Cideciyan AV, Jacobson SG, Beltran WA, et al. Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Proc Natl Acad Sci U S A 2013;110(6):E517-E525.
4. Jacobson SG, Cideciyan AV, Roman AJ, et al. Improvement and decline in vision with gene therapy in childhood blindness. N Engl J Med 2015;372:1920-6.
5. Bainbridge J W B, Mehat MS, Sundaram V, et al. Long-term effect of gene therapy on Leber's congenital amaurosis. N Engl J Med 2015;372:1887-97.
6. Cideciyan AV, Aleman TS, Boye SL, et al. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A 2008;105:15112-7.
7. Acland GM, Aguirre GD, Bennett J, et al. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. Mol Ther 2005;12:1072-82.
8. Rohrer B, Lohr HR, Humphries P, Redmond TM, Seeliger MW, Crouch RK. Cone opsin mislocalization in Rpe65-/-mice: a defect that can be corrected by 11-cis retinal. Invest Ophthalmol Vis Sci 2005;46:3876-82.
9. Bramall AN, Wright AF, Jacobson SG, McInnes RR. The genomic, biochemical, and cellular responses of the retina in inherited photoreceptor degenerations and prospects for the treatment of these disorders. Annu Rev Neurosci 2010;33:441-72.
10. Mowat FM, Gornik KR, Dinculescu A, et al. Tyrosine capsidmutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach. Gene Ther 2014;21:96-105.