Linked Life Data

21142470

Source:http://linkedlifedata.com/resource/pubmed/id/21142470

Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
5
pubmed:dateCreated
2011-4-27
pubmed:abstractText
Gene therapy studies in primates can provide important information regarding vector tropism, specific cellular expression, biodistribution, and safety prior to clinical trials. In this study, we report the assessment of transduction efficiency of recombinant adeno-associated virus (rAAV) vectors using human postmortem retina. Transductions were performed using two in vitro models prepared from human tissue: dissociated cell cultures and retinal explants. These models were used to assess cellular tropism and selectivity of rAAV vectors encoding for fluorescent proteins under the control of different promoters. These promoters were a ubiquitous cytomegalovirus promoter and a cell type-specific promoter targeting expression in ON bipolar cells. The results demonstrate that this in vitro approach can limit the use of living primates for the validation of gene therapy in vision and ophthalmology.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:status
MEDLINE
pubmed:month
May
pubmed:issn
1557-7422
pubmed:author
pubmed:issnType
Electronic
pubmed:volume
22
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
587-93
pubmed:meshHeading
pubmed:year
2011
pubmed:articleTitle
Gene therapy in ophthalmology: validation on cultured retinal cells and explants from postmortem human eyes.
pubmed:affiliation
INSERM, U968, Paris, 75 012, France.
pubmed:publicationType
Journal Article, In Vitro, Research Support, Non-U.S. Gov't