PubRank

Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

Clinical Trial ID NCT00821340

PubWeight™ 8.51‹?›

🔗 Visit the ClinicalTrials.gov page for NCT00821340

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 Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 2008 15.30
3 Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther 2008 8.83
4 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
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 Viral vectors for neurotrophic factor delivery: a gene therapy approach for neurodegenerative diseases of the CNS. Pharmacol Res 2009 1.20
7 Preliminary results of gene therapy for retinal degeneration. N Engl J Med 2008 1.14
8 Drug and gene delivery to the back of the eye: from bench to bedside. Invest Ophthalmol Vis Sci 2014 0.93
9 Mid-stage intervention achieves similar efficacy as conventional early-stage treatment using gene therapy in a pre-clinical model of retinitis pigmentosa. Hum Mol Genet 2013 0.91
10 Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium. Invest Ophthalmol Vis Sci 2015 0.88
11 Promising and delivering gene therapies for vision loss. Vision Res 2014 0.85
12 Dog models for blinding inherited retinal dystrophies. Hum Gene Ther Clin Dev 2015 0.80
13 Let There Be Light: Gene and Cell Therapy for Blindness. Hum Gene Ther 2016 0.79
14 Recombinant adeno-associated virus vectors in the treatment of rare diseases. Expert Opin Orphan Drugs 2015 0.75
15 Ocular gene transfer in the spotlight: implications of newspaper content for clinical communications. BMC Med Ethics 2014 0.75
16 Development of gene and stem cell therapy for ocular neurodegeneration. Int J Ophthalmol 2015 0.75
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