Evidence map›Paper›PMID 41624335›Full record

ArticleMolecular therapy. Nucleic acids2026

Upregulation of a CFTR mRNA isoform has therapeutic potential for the treatment of 3' CFTR PTC variants.

Normand E Allaire, Matthew S Armstrong, Jae Seok Yoon, Mercy C Walker, Priyanka Bhatt, Jan M Harrington, Yi Cheng, Eric I Wong, John E Mahoney, Hillary C Valley and 6 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 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

16 authors.

Normand E AllaireCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
Matthew S ArmstrongCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
Jae Seok YoonCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
Mercy C WalkerCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
Priyanka BhattCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
Jan M HarringtonCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
Yi ChengCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
Eric I WongCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
John E MahoneyCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
Hillary C ValleyCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
Caitlin M MacadinoCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
Hermann J BihlerCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
Andrey Y SivachenkoCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
Calvin U CottonCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
John P CarulliCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.
Martin MenseCFFT Lab, Cystic Fibrosis Foundation, Lexington, MA 02421, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Nonsense or premature termination codon (PTC) variants of the CFTR gene are pathogenic and found in ∼10% of North American people with cystic fibrosis. In addition to encoding incomplete proteins, PTC variants induce nonsense-mediated mRNA decay (NMD), leading to ∼80%-90% reduction in full-length mRNA. This reduction is a contributor to PTC mutation-related pathology. E22 trunc is a naturally occurring truncated CFTR mRNA that terminates before the W1282X PTC variant and is resistant to NMD. To induce its expression, antisense oligonucleotides (ASOs) were tiled across intron 22 splice donor (SD) and splice acceptor (SA) sites. Top SD/SA ASO pairs were assessed for their impact on e22 trunc mRNA, e22 trunc protein, and CFTR-mediated chloride (Cl

Indexed as

alternative polyadenylationantisense oligonucleotideCFTRcystic fibrosismRNA isoformmRNA processingMT: Oligonucleotides: Therapies and ApplicationsW1282X

Identifiers

PMID41624335
PMCPMC12858342

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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.