ArticleGene therapy2010
AAV-mediated knockdown of peripherin-2 in vivo using miRNA-based hairpins.
Article in Gene therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
28 citing papers in PubMed, 53 citations in OpenAlex.
- Cell-specific regulation of gene expression using splicing-dependent frameshifting.Nature communications · 2022Article
- MicroRNAs as therapeutic targets in breast cancer metastasis.Drug delivery and translational research · 2022Review
- An Analysis of Mechanisms for Cellular Uptake of miRNAs to Enhance Drug Delivery and Efficacy in Cancer Chemoresistance.Non-coding RNA · 2021Review
- Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction.Frontiers in immunology · 2021Article
- ROM1 contributes to phenotypic heterogeneity in PRPH2-associated retinal disease.Human molecular genetics · 2020Article
- Review
- The Peripherin Gene Regulates the Migration of Bone Marrow Mesenchymal Stem Cells in Wuzhishan Mini Pigs.Stem cells international · 2020Article
- Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity.JCI insight · 2019Article
- Novel stem cell and gene therapy in diabetic retinopathy, age related macular degeneration, and retinitis pigmentosa.International journal of retina and vitreous · 2019Review
- Improving miRNA Delivery by Optimizing miRNA Expression Cassettes in Diverse Virus Vectors.Human gene therapy methods · 2017Review
- A High-Throughput Drug Screening Strategy for Detecting Rhodopsin P23H Mutant Rescue and Degradation.Investigative ophthalmology & visual science · 2015Article
- RNA interference-based therapy for spinocerebellar ataxia type 7 retinal degeneration.PloS one · 2014Article
- Non-coding RNAs in the development of sensory organs and related diseases.Cellular and molecular life sciences : CMLS · 2013Review
- Mutation-independent rescue of a novel mouse model of Retinitis Pigmentosa.Gene therapy · 2013Article
- Gene therapy for retinal disease.Translational research : the journal of laboratory and clinical medicine · 2013Review
- A comprehensive review of retinal gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2013Review
- Artificial microRNA interference targeting AT(1a) receptors in paraventricular nucleus attenuates hypertension in rats.Gene therapy · 2012Article
- Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice.Human gene therapy · 2012Article
- Gene-based therapies for dominantly inherited retinopathies.Gene therapy · 2012Review
- Gene delivery to the retina: from mouse to man.Methods in enzymology · 2012Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors at 1 institution in 1 country.
Funding
Abstract
Gene therapy for inherited retinal degeneration in which expression of a mutant allele has a gain-of-function effect on photoreceptor cells is likely to depend on efficient silencing of the mutated allele. Peripherin-2 (Prph2, also known as peripherin/RDS) is an abundantly expressed photoreceptor-specific gene. In humans, gain-of-function mutations in PRPH2 result in both autosomal dominant retinitis pigmentosa and dominant maculopathies. Gene-silencing strategies for these conditions include RNA interference by short hairpin RNAs (shRNAs). Recent evidence suggests that microRNA (miRNA)-based hairpins may offer a safer and more effective alternative. In this study, we used for the first time a virally transferred miRNA-based hairpin to silence Prph2 in the murine retina. The results show that an miRNA-based shRNA can efficiently and specifically silence Prph2 in vivo as early as 3 weeks after AAV2/8-mediated subretinal delivery, leading to a nearly 50% reduction of photoreceptor cells after 5 weeks. We conclude that miRNA-based hairpins can achieve rapid and robust gene silencing after efficient vector-mediated delivery to the retina. The rationale of using an miRNA-based template to improve the silencing efficiency of a hairpin may prove valuable for allele-specific silencing in which the choice for an RNAi target is limited and offers an alternative therapeutic strategy for the treatment of dominant retinopathies.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.