Source:http://linkedlifedata.com/resource/pubmed/id/18774912
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rdf:type | |
lifeskim:mentions |
umls-concept:C0017337,
umls-concept:C0026882,
umls-concept:C0086022,
umls-concept:C0087111,
umls-concept:C0339527,
umls-concept:C0442335,
umls-concept:C0678226,
umls-concept:C0920321,
umls-concept:C1274040,
umls-concept:C1299003,
umls-concept:C1419622,
umls-concept:C1533685,
umls-concept:C1564874,
umls-concept:C1705099,
umls-concept:C2699708
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pubmed:issue |
10
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pubmed:dateCreated |
2009-3-30
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pubmed:abstractText |
Leber congenital amaurosis (LCA) is a group of autosomal recessive blinding retinal diseases that are incurable. One molecular form is caused by mutations in the RPE65 (retinal pigment epithelium-specific 65-kDa) gene. A recombinant adeno-associated virus serotype 2 (rAAV2) vector, altered to carry the human RPE65 gene (rAAV2-CBSB-hRPE65), restored vision in animal models with RPE65 deficiency. A clinical trial was designed to assess the safety of rAAV2-CBSB-hRPE65 in subjects with RPE65-LCA. Three young adults (ages 21-24 years) with RPE65-LCA received a uniocular subretinal injection of 5.96 x 10(10) vector genomes in 150 microl and were studied with follow-up examinations for 90 days. Ocular safety, the primary outcome, was assessed by clinical eye examination. Visual function was measured by visual acuity and dark-adapted full-field sensitivity testing (FST); central retinal structure was monitored by optical coherence tomography (OCT). Neither vector-related serious adverse events nor systemic toxicities were detected. Visual acuity was not significantly different from baseline; one patient showed retinal thinning at the fovea by OCT. All patients self-reported increased visual sensitivity in the study eye compared with their control eye, especially noticeable under reduced ambient light conditions. The dark-adapted FST results were compared between baseline and 30-90 days after treatment. For study eyes, sensitivity increases from mean baseline were highly significant (p < 0.001); whereas, for control eyes, sensitivity changes were not significant (p = 0.99). Comparisons are drawn between the present work and two other studies of ocular gene therapy for RPE65-LCA that were carried out contemporaneously and reported.
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pubmed:grant | |
pubmed:language |
eng
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pubmed:journal | |
pubmed:citationSubset |
IM
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pubmed:chemical | |
pubmed:status |
MEDLINE
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pubmed:month |
Oct
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pubmed:issn |
1557-7422
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pubmed:author |
pubmed-author:AlemanTomas STS,
pubmed-author:BoyeSanford LSL,
pubmed-author:ByrneBarry JBJ,
pubmed-author:CideciyanArtur VAV,
pubmed-author:ConlonThomas JTJ,
pubmed-author:FlotteTerence RTR,
pubmed-author:HauswirthWilliam WWW,
pubmed-author:JacobsonSamuel GSG,
pubmed-author:KaushalShaleshS,
pubmed-author:SchwartzSharon BSB,
pubmed-author:WangLiliL
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pubmed:issnType |
Electronic
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pubmed:volume |
19
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
979-90
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pubmed:dateRevised |
2010-9-24
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pubmed:meshHeading |
pubmed-meshheading:18774912-Adolescent,
pubmed-meshheading:18774912-Adult,
pubmed-meshheading:18774912-Animals,
pubmed-meshheading:18774912-Blindness,
pubmed-meshheading:18774912-Carrier Proteins,
pubmed-meshheading:18774912-Dark Adaptation,
pubmed-meshheading:18774912-Dependovirus,
pubmed-meshheading:18774912-Eye Proteins,
pubmed-meshheading:18774912-Female,
pubmed-meshheading:18774912-Follow-Up Studies,
pubmed-meshheading:18774912-Gene Therapy,
pubmed-meshheading:18774912-Genetic Diseases, Inborn,
pubmed-meshheading:18774912-Genetic Vectors,
pubmed-meshheading:18774912-Humans,
pubmed-meshheading:18774912-Male,
pubmed-meshheading:18774912-Mutation,
pubmed-meshheading:18774912-Retina,
pubmed-meshheading:18774912-Vision, Ocular
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pubmed:year |
2008
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pubmed:articleTitle |
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.
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pubmed:affiliation |
Department of Ophthalmology, University of Florida, Gainesville, FL 32610, USA.
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pubmed:publicationType |
Journal Article,
Comparative Study,
Clinical Trial, Phase I,
Research Support, N.I.H., Extramural
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