Evidence map›Paper›PMID 40436630›Full record

ArticleRNA (New York, N.Y.)2025

Absolute quantification of mammalian microRNAs for therapeutic RNA cleavage and detargeting.

Carolyn Kraus, Jiayi Wang, Haiying Zheng, Jennifer Broderick, Nandagopal Ajaykumar, Mina Zamani, Mengqi Yang, Katharine Cecchini, Shun-Qing Liang, Olena Kolumba and 5 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in RNA (New York, N.Y.), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. Review
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

15 authors.

Carolyn Kraus *RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts 01655, USA.
Jiayi Wang *RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts 01655, USA.
Haiying ZhengDepartment of Biology, New York University, New York, New York 10003, USA.
Jennifer BroderickRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts 01655, USA.
Nandagopal AjaykumarDepartment of Biology, New York University, New York, New York 10003, USA.
Mina ZamaniDepartment of Biology, New York University, New York, New York 10003, USA.
Mengqi YangRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts 01655, USA.
Katharine CecchiniRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts 01655, USA.
Shun-Qing LiangDivision of Gastroenterology, Hepatology, and Nutrition, University of Minnesota Twin Cities, Minneapolis, Minnesota 55455, USA.
Olena KolumbaDepartment of Biology, New York University, New York, New York 10003, USA.
Kathryn ChaseRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts 01655, USA.
Jooyoung LeeRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts 01655, USA.
Wen XueRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts 01655, USA.
Erik J SontheimerRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts 01655, USA.ORCID 0000-0002-0881-0310
Ildar GainetdinovDepartment of Biology, New York University, New York, New York 10003, USA ildar.gainetdinov@nyu.edu.

Funding

In vivo prime editing for precision cancer mouse modelsR01CA275945 · NCI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Wen Xue · 2023 to 2026
$2.5M
Advanced Delivery Platforms for Base Editing In VivoR01GM150273 · NIGMS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI ERIK J. SONTHEIMER, Wen Xue · 2023 to 2026
$2.4M
The Quantitative Biological Systems Training (QBIST) ProgramT32GM132037 · NIGMS · NEW YORK UNIVERSITY · PI David Gresham, Christine Vogel · 2019 to 2026
$2.3M
Muscle-Specific CRISPR/Cas9 Exon Skipping for Duchenne Muscular Dystrophy TherapeuticsF31AR082678 · NIAMS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI KRAUS, CAROLYN · 2023 to 2024
$66k
NCI NIH HHS R01 CA275945NIAMS NIH HHS F31 AR082678NIGMS NIH HHS R01 GM150273NIGMS NIH HHS T32 GM132037
6 · The paper itself

Abstract

MicroRNAs (miRNAs) are small regulatory RNAs that destabilize partially complementary transcripts and cleave perfectly paired targets. miRNAs are often expressed in a specific tissue, allowing miRNA-directed cleavage to be used to prevent genome editing or gene replacement therapy in unintended cell types, a strategy called detargeting. miRNA intracellular concentration influences the potency of gene silencing, yet the absolute steady-state levels of just a few miRNAs have been determined in mammalian tissues. Here, we report the absolute abundance of miRNAs in 14 human and mouse cell lines and 17 mouse tissues, including eight brain regions. Our experiments in human cultured cells demonstrate that both miRNA and target levels influence efficacy of cleavage of fully complementary transcripts. We report the miRNA concentration required for productive cleavage of highly expressed transcripts and identify mouse miRNAs that reach this threshold in vivo.

Indexed as

MicroRNAsRNA CleavageAnimalsBrainCell LineHumansMiceMicroRNAsabsolute abundancedetargetingmicroRNAtissue-specific expression

Identifiers

PMID40436630
PMCPMC12265943

What OpenQuestion holds

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LicenceCC BY
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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.