Evidence map›Paper›PMID 42901161›Full record

ArticleMolecular cell2026

Regulatable in vivo gene expression via adaptamers.

Jack Bryant, Laura Herron, Yesh Doctor, Finn McRae, Jocelyn Wang, Theodore Hirsch, Andrew Portell, Satheesh Kumar, Prashant Mali

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular cell, 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

9 authors.

Jack BryantDepartment of Bioengineering, University of California San Diego, La Jolla, CA, USA.
Laura HerronDepartment of Bioengineering, University of California San Diego, La Jolla, CA, USA.
Yesh DoctorDepartment of Bioengineering, University of California San Diego, La Jolla, CA, USA.
Finn McRaeDepartment of Bioengineering, University of California San Diego, La Jolla, CA, USA.
Jocelyn WangDepartment of Bioengineering, University of California San Diego, La Jolla, CA, USA.
Theodore HirschDepartment of Bioengineering, University of California San Diego, La Jolla, CA, USA.
Andrew PortellDepartment of Bioengineering, University of California San Diego, La Jolla, CA, USA.
Satheesh KumarDepartment of Bioengineering, University of California San Diego, La Jolla, CA, USA.
Prashant MaliDepartment of Bioengineering, University of California San Diego, La Jolla, CA, USA. Electronic address: pmali@ucsd.edu.

Funding

Next generation massively multiplexed combinatorial genetic screensR01HG012351 · NHGRI · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI Trey Ideker, Prashant Mali · 2023 to 2026
$2.7M
Systematic functional mapping and interrogation of oncogenic intrinsically disordered regionsR01CA310063 · NCI · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI Hannah Kathryn Carter, Prashant Mali · 2026 to 2026
$662k
NCI NIH HHS R01 CA310063NHGRI NIH HHS R01 HG012351
6 · The paper itself

Abstract

Precise, reversible control of transgene expression is essential for safe and durable gene therapy, yet current inducible systems remain difficult to translate in vivo due to large size, limited induction duration, and dependence on immunogenic regulators. Here, we present the adaptamer (adenosine deaminases acting on RNA [ADAR]-modulatable aptamer), a compact (<120 base pair) RNA switch that couples FDA-approved small-molecule-responsive aptamers with endogenous ADAR-mediated RNA editing to regulate expression. Ligand binding stabilizes a double-stranded RNA structure that recruits ADAR to convert a stop codon into a sense codon, restoring downstream protein translation. This cleavage-free, post-transcriptional mechanism enables precise, small-molecule-dependent modulation without exogenous protein machinery. We demonstrate that adaptamers are highly functional across multiple cell lines, including human T cells. In mice, adeno-associated virus (AAV)-delivered adaptamer-controlled FGF21 expression induced metabolic remodeling, reversing obesity and hepatic steatosis. This minimal, programmable system offers a clinically compatible approach for tunable genetic medicines and safe deployment of pleiotropic and dose-limited proteins.

Indexed as

adaptamerADARaptamerFGF21gene therapyinducible expressionorthogonal controlriboswitchRNA editingRNA engineering

Identifiers

PMID42901161
PMCPMC13692725

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.