Safety Study in Subjects With Leber Congenital Amaurosis

Clinical Trial ID NCT00516477

PubWeight™ 64.75‹?›

🔗 Visit the page for NCT00516477

Top papers

Rank Title Journal Year PubWeight™‹?›
1 Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 2008 17.21
2 Gene therapy restores vision in a canine model of childhood blindness. Nat Genet 2001 7.88
3 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 7.16
4 Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 2009 6.71
5 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 4.74
6 Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet 2011 4.67
7 Photoreceptor layer topography in children with leber congenital amaurosis caused by RPE65 mutations. Invest Ophthalmol Vis Sci 2008 2.06
8 Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina. Proc Natl Acad Sci U S A 1999 1.89
9 Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2. Ophthalmology 2013 1.85
10 Retinitis pigmentosa: genes and disease mechanisms. Curr Genomics 2011 1.85
11 Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy. Prog Retin Eye Res 2010 1.81
12 A comprehensive review of retinal gene therapy. Mol Ther 2013 1.70
13 The human visual cortex responds to gene therapy-mediated recovery of retinal function. J Clin Invest 2011 1.52
14 RPE65: role in the visual cycle, human retinal disease, and gene therapy. Ophthalmic Genet 2009 1.39
15 Viral vectors for neurotrophic factor delivery: a gene therapy approach for neurodegenerative diseases of the CNS. Pharmacol Res 2009 1.20
16 Gene therapy for retinal disease. Transl Res 2013 1.03
17 Pupillometric analysis for assessment of gene therapy in Leber Congenital Amaurosis patients. Biomed Eng Online 2012 0.98
18 Nanoparticle-mediated gene transfer specific to retinal pigment epithelial cells. Biomaterials 2011 0.96
19 Vector platforms for gene therapy of inherited retinopathies. Prog Retin Eye Res 2014 0.94
20 MicroRNA-restricted transgene expression in the retina. PLoS One 2011 0.92
21 Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients. Hum Gene Ther 2012 0.90
22 Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium. Invest Ophthalmol Vis Sci 2015 0.88
23 fMRI of retina-originated phosphenes experienced by patients with Leber congenital amaurosis. PLoS One 2014 0.88
24 Gene therapy of inherited retinal degenerations: prospects and challenges. Hum Gene Ther 2015 0.87
25 Plasticity of the human visual system after retinal gene therapy in patients with Leber's congenital amaurosis. Sci Transl Med 2015 0.86
26 Promising and delivering gene therapies for vision loss. Vision Res 2014 0.85
27 Prospectives for gene therapy of retinal degenerations. Curr Genomics 2012 0.83
28 Cellular unfolded protein response against viruses used in gene therapy. Front Microbiol 2014 0.83
29 Dog models for blinding inherited retinal dystrophies. Hum Gene Ther Clin Dev 2015 0.80
30 Improving clinical efficacy of adeno associated vectors by rational capsid bioengineering. J Biomed Sci 2014 0.80
31 Let There Be Light: Gene and Cell Therapy for Blindness. Hum Gene Ther 2016 0.79
32 Non-viral therapeutic approaches to ocular diseases: An overview and future directions. J Control Release 2015 0.78
33 Retinal gene therapy using adeno-associated viral vectors: multiple applications for a small virus. Hum Gene Ther 2014 0.77
34 Ocular gene transfer in the spotlight: implications of newspaper content for clinical communications. BMC Med Ethics 2014 0.75
35 Development of gene and stem cell therapy for ocular neurodegeneration. Int J Ophthalmol 2015 0.75
36 Recombinant adeno-associated virus vectors in the treatment of rare diseases. Expert Opin Orphan Drugs 2015 0.75
Next 100