Design and Validation of Mirtron-Mediated RNA Interference for Gene Therapy.

Qingyun Zheng, Harry O Orlans, Federica Staurenghi, Michelle E McClements, Robert E MacLaren

Abstract read

In one paragraph

Article in Methods in molecular biology (Clifton, N.J.), 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it

0cells of the map it votes in

0citing papers in PubMed

–field-weighted citation impact

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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.

Qingyun ZhengNuffield Department of Clinical Neurosciences & NIHR Oxford Biomedical Research Centre, University of Oxford, UK and Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK.

Harry O OrlansNuffield Department of Clinical Neurosciences & NIHR Oxford Biomedical Research Centre, University of Oxford, UK and Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK.

Federica StaurenghiNuffield Department of Clinical Neurosciences & NIHR Oxford Biomedical Research Centre, University of Oxford, UK and Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK.

Michelle E McClementsNuffield Department of Clinical Neurosciences & NIHR Oxford Biomedical Research Centre, University of Oxford, UK and Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK.

Robert E MacLarenNuffield Department of Clinical Neurosciences & NIHR Oxford Biomedical Research Centre, University of Oxford, UK and Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK. maclaren@eye.ox.ac.uk.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Mirtrons, a subclass of intronic microRNAs, offer an alternative RNA interference (RNAi) mechanism by bypassing Drosha processing. This protocol details the design and production of mirtron-based constructs aimed at achieving efficient gene knockdown. By embedding mirtrons in the 5' untranslated region (UTR) of a transgene, we evaluated processing efficiency and RNAi activity. The compact size of mirtrons facilitates concurrent gene knockdown and supplementation within a single adeno-associated virus (AAV) vector. Our findings present a novel strategy for gene regulation in therapeutic and research applications.

Indexed as

Genetic TherapyMicroRNAsRNA Interference5' Untranslated RegionsDependovirusGene Knockdown TechniquesGenetic VectorsHumansIntrons5' Untranslated RegionsMicroRNAsDNA cloningGene therapyMirtronRNAi

Identifiers

PMID42020911

DOI10.1038/s41467-021-25204-3

What OpenQuestion holds

Textmetadata

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.