2011
DOI: 10.1371/journal.pone.0022166
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MicroRNA-Restricted Transgene Expression in the Retina

Abstract: BackgroundGene transfer using adeno-associated viral (AAV) vectors has been successfully applied in the retina for the treatment of inherited retinal dystrophies. Recently, microRNAs have been exploited to fine-tune transgene expression improving therapeutic outcomes. Here we evaluated the ability of retinal-expressed microRNAs to restrict AAV-mediated transgene expression to specific retinal cell types that represent the main targets of common inherited blinding conditions.Methodology/Principal FindingsTo thi… Show more

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Cited by 58 publications
(54 citation statements)
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“…We then tested the rod and cone PR transduction of AAV2/8 in combination with (1) the ubiquitous CMV promoter; (2) the PR-specific GRK1 promoter; and (3) the CMV promoter in combination with miR204T, which we had previously demonstrated to restrict AAV-mediated transgene expression to the ONL in mice and pigs (Karali et al, 2011). We therefore subretinally injected the various AAV2/ 8 vectors encoding for EGFP (1 · 10 10 GC/eye of each vector) in 11-week-old female LW pigs (n = 3 eyes/vector).…”
Section: Resultsmentioning
confidence: 99%
See 2 more Smart Citations
“…We then tested the rod and cone PR transduction of AAV2/8 in combination with (1) the ubiquitous CMV promoter; (2) the PR-specific GRK1 promoter; and (3) the CMV promoter in combination with miR204T, which we had previously demonstrated to restrict AAV-mediated transgene expression to the ONL in mice and pigs (Karali et al, 2011). We therefore subretinally injected the various AAV2/ 8 vectors encoding for EGFP (1 · 10 10 GC/eye of each vector) in 11-week-old female LW pigs (n = 3 eyes/vector).…”
Section: Resultsmentioning
confidence: 99%
“…For the production of AAV encoding EGFP with different promoters, pAAV2.1-CMV-EGFP (Allocca et al, 2007), pAAV2.1-CMV-EGFP-4XmiR204T (Karali et al, 2011), and pAAV2.1-GRK1-EGFP (Allocca et al, 2007) plasmids were used for the production of AAV2/8 vectors. The GRK1 promoter was previously described (Young et al, 2003) and includes the -112 to + 86 nucleotides from the promoter region of the human GRK1 gene (1810011-1810209 of GenBank ID: NW_004078078.1).…”
Section: Methodsmentioning
confidence: 99%
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“…97 These and other studies indicate the superiority of miRNA-based AAV approaches for some therapeutic applications with respect to the efficiency and safety. [98][99][100][101] …”
Section: 45mentioning
confidence: 99%
“…[86][87][88] Recently, a comprehensive atlas of the complete mouse eye, including the retina, has been developed, 89 and a tool for miRrestricted transgene expression in the retina has been set up. 90 Until now, evidence for a miR signature in hypertensive retinopathy is lacking, yet the premise of miR-oriented research on this topic could be promising.…”
Section: Studies In Other Models Of Kidney Diseasementioning
confidence: 99%