Clinical Trial
doi: 10.1056/NEJMoa0802268. Epub 2008 Apr 27.Effect of gene therapy on visual function in Leber's congenital amaurosis
James W B Bainbridge 1, Alexander J Smith, Susie S Barker, Scott Robbie, Robert Henderson, Kamaljit Balaggan, Ananth Viswanathan, Graham E Holder, Andrew Stockman, Nick Tyler, Simon Petersen-Jones, Shomi S Bhattacharya, Adrian J Thrasher, Fred W Fitzke, Barrie J Carter, Gary S Rubin, Anthony T Moore, Robin R Ali
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- PMID:18441371
- DOI: 10.1056/NEJMoa0802268
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Clinical Trial
Effect of gene therapy on visual function in Leber's congenital amaurosis
James W B Bainbridge et al. N Engl J Med..
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Abstract
Early-onset, severe retinal dystrophy caused by mutations in the gene encoding retinal pigment epithelium-specific 65-kD protein (RPE65) is associated with poor vision at birth and complete loss of vision in early adulthood. We administered to three young adult patients subretinal injections of recombinant adeno-associated virus vector 2/2 expressing RPE65 complementary DNA (cDNA) under the control of a human RPE65 promoter. There were no serious adverse events. There was no clinically significant change in visual acuity or in peripheral visual fields on Goldmann perimetry in any of the three patients. We detected no change in retinal responses on electroretinography. One patient had significant improvement in visual function on microperimetry and on dark-adapted perimetry. This patient also showed improvement in a subjective test of visual mobility. These findings provide support for further clinical studies of this experimental approach in other patients with mutant RPE65. (ClinicalTrials.gov number,NCT00643747 [ClinicalTrials.gov].).
Copyright 2008 Massachusetts Medical Society.
Comment in
- Preliminary results of gene therapy for retinal degeneration.Miller JW.Miller JW.N Engl J Med. 2008 May 22;358(21):2282-4. doi: 10.1056/NEJMe0803081. Epub 2008 Apr 27.N Engl J Med. 2008.PMID:18441372No abstract available.
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