Evidence map›Paper›PMID 30290306›Full record

ArticleMolecular therapy. Nucleic acids2018

Protecting Pax6 3' UTR from MicroRNA-7 Partially Restores PAX6 in Islets from an Aniridia Mouse Model.

Kevin Yongblah, Spencer C Alford, Bridget C Ryan, Robert L Chow, Perry L Howard

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Nucleic acids, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
0.6field-weighted citation impact, top 37% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

7 citing papers in PubMed, 13 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors at 1 institution in 1 country.

Kevin YongblahDepartment of Biochemistry and Microbiology, University of Victoria, P.O. Box 1700 STN CSC, Victoria, BC V8W2Y2, Canada.
Spencer C AlfordDepartment of Biochemistry and Microbiology, University of Victoria, P.O. Box 1700 STN CSC, Victoria, BC V8W2Y2, Canada.
Bridget C RyanDepartment of Biology, University of Victoria, P.O. Box 1700 STN CSC, Victoria, BC V8W 2Y2, Canada.
Robert L ChowDepartment of Biology, University of Victoria, P.O. Box 1700 STN CSC, Victoria, BC V8W 2Y2, Canada.
Perry L HowardDepartment of Biochemistry and Microbiology, University of Victoria, P.O. Box 1700 STN CSC, Victoria, BC V8W2Y2, Canada. Electronic address: phoward@uvic.ca.
University of Victoria · CA

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Aniridia is a rare congenital syndrome that is associated with reduced visual acuity and progressive loss of vision. Aniridia patients may also develop systemic health issues associated with defects in the pancreas, digestive, and central nervous systems. The spectrum of symptoms associated with aniridia is due to haploinsufficiency of the paired box 6 gene (PAX6) and its role in the development and maintenance of the affected tissues. Here, we isolated pancreatic islets from mice heterozygous for Pax6 to test whether a Pax6-specific miRNA suppression (target protector) strategy can restore PAX6 protein levels. We show that miR-7 and miR-375 target specific sites within the Pax6 3' UTR in a mouse pancreatic β-insulinoma cell line. Tough decoys (Tuds) against miR-7 and miR-375 increase expression of a mouse Pax6 3' UTR luciferase reporter and increase PAX6 protein levels in these cells. Finally, we demonstrate that the shielding of the miR-7 binding site with a target protector restores PAX6 protein levels in the Pax6 heterozygous islets. The data presented here represent a proof of concept for RNA-based therapy for the progressive defects associated with aniridia and suggest the target protector approach may be a useful therapeutic strategy for other haploinsufficiency diseases.

Indexed as

aniridiaisletsmicroRNAPax6target protector

Identifiers

PMID30290306
PMCPMC6171161
OpenAlexW2888859121

What OpenQuestion holds

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LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.