Evidence map›Paper›PMID 41184552›Full record

ArticleNature methods2025

A portable poison exon for small-molecule control of mammalian gene expression.

Qian Hou, Maxim Oleynikov, Xiaohan Mei, Linghao Dong, Timo Hagen, Samie R Jaffrey

Abstract read
In one paragraph

Article in Nature methods, 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. Regulatable In Vivo Gene Expression via Adaptamers.bioRxiv : the preprint server for biology · 2025
    Article
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

6 authors.

Qian HouDepartment of Pharmacology, Weill Cornell Medical College, Cornell University, New York, NY, USA.ORCID http://orcid.org/0000-0003-1872-7964
Maxim OleynikovDepartment of Pharmacology, Weill Cornell Medical College, Cornell University, New York, NY, USA.ORCID http://orcid.org/0000-0002-6892-9743
Xiaohan MeiDepartment of Pharmacology, Weill Cornell Medical College, Cornell University, New York, NY, USA.ORCID http://orcid.org/0000-0003-4677-4653
Linghao DongDepartment of Pharmacology, Weill Cornell Medical College, Cornell University, New York, NY, USA.ORCID http://orcid.org/0009-0001-2725-1868
Timo HagenDepartment of Pharmacology, Weill Cornell Medical College, Cornell University, New York, NY, USA.
Samie R JaffreyDepartment of Pharmacology, Weill Cornell Medical College, Cornell University, New York, NY, USA. srj2003@med.cornell.edu.ORCID http://orcid.org/0000-0003-3615-6958

Funding

Center for Multi-Scale Analysis of the Human EpitranscriptomeRM1HG011563 · NHGRI · WEILL MEDICAL COLL OF CORNELL UNIV · PI SAMIE R JAFFREY, Kathryn D Meyer · 2021 to 2026
$22.2M
New mechanisms and technologies for understanding post-transcriptional gene regulation in neuronsR35NS111631 · NINDS · WEILL MEDICAL COLL OF CORNELL UNIV · PI SAMIE R JAFFREY · 2019 to 2026
$8.6M
Ultra-sensitive multi-mode laser-scanning imaging systemS10OD030335 · OD · WEILL MEDICAL COLL OF CORNELL UNIV · PI JAFFREY, SAMIE R · 2021 to 2021
$163k
The role of heme in the oxidative modification of mRNAF31NS139612 · NINDS · WEILL MEDICAL COLL OF CORNELL UNIV · PI Maxim Oleynikov · 2025 to 2026
$100k
NHGRI NIH HHS RM1 HG011563NIH HHS S10 OD030335NINDS NIH HHS R35 NS111631ODCDC CDC HHS S10 OD030335Swiss National Science Foundation | nccr - on the move (National Center of Competence in Research - The Migration-Mobility Nexus) 199851U.S. Department of Health & Human Services | NIH | National Institute of General Medical Sciences (NIGMS) NIH T32 GM115327U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke (NINDS) F31NS139612U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke (NINDS) R35NS111631
6 · The paper itself

Abstract

The ability to precisely control gene expression using small-molecule drugs is a valuable tool in research and has important therapeutic potential. However, existing systems are often limited by the toxicity of the drugs and the need to alter gene sequences or endogenous regulatory elements. Here, we introduce Cyclone (acyclovir-controlled poison exon), an acyclovir-controlled poison exon cassette that can be used for small-molecule control of both transgene and endogenous gene expression. Cyclone is a portable 'intron-poison exon-intron' element that can be inserted into nearly any gene and is completely removed upon acyclovir treatment, leaving the native transcript intact. Cyclone offers tunable, reversible gene expression with nearly undetectable background and a ~295-fold activation. We also present Pac-Cyclone, a cassette that simplifies the generation of cell lines with acyclovir-controlled endogenous gene expression. Finally, we demonstrate the programmability of Cyclone, underscoring its potential for developing diverse genetic circuits controlled by various ligands.

Indexed as

AcyclovirExonsGene ExpressionGene Expression RegulationAnimalsHEK293 CellsHumansAcyclovir

Identifiers

PMID41184552
PMCPMC13102273

What OpenQuestion holds

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Registered trials

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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.