Evidence map›Paper›PMID 36658041›Full record

ArticleJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2023

Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis.

Normand E Allaire, Uta Griesenbach, Batsheva Kerem, John D Lueck, Noemie Stanleigh, Yifat S Oren

Abstract read
In one paragraph

Article in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing 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

12 citing papers in PubMed.

  1. Article
  2. Review
  3. Engineered cargo-free nanoparticle decoys for phagocytic modulation of macrophages.Journal of controlled release : official journal of the Controlled Release Society · 2026
    Article
  4. Article
  5. Review
  6. Review
  7. Review
  8. Review
  9. Lentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-in-Human Trial.American journal of respiratory and critical care medicine · 2024
    Review
  10. A cystic fibrosis gene editing approach that is on target.Molecular therapy. Nucleic acids · 2024
    Article
  11. Review
  12. 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

6 authors.

Normand E AllaireCystic Fibrosis Foundation Laboratory, Lexington MA, USA. Electronic address: nallaire@cff.org.
Uta GriesenbachNational Heart and Lung Institute, Imperial College London and the UK Respiratory Gene Therapy Consortium, UK.
Batsheva KeremDepartment of Genetics, The Life Sciences Institute, Hebrew University, Jerusalem, Israel.
John D LueckDepartment of Pharmacology and Physiology, University of Rochester Medical Center, Rochester, NY, USA.
Noemie StanleighDepartment of Genetics, The Life Sciences Institute, Hebrew University, Jerusalem, Israel.
Yifat S OrenSpliSenseTherapeutics, Biohouse Labs, Haddasah Ein Karem, Jerusalem, IL.

Funding

In vivo delivery of engineered tRNAs for suppression of nonsense mutationsR01HL153988 · NHLBI · UNIVERSITY OF ROCHESTER · PI John D. Lueck · 2021 to 2026
$3.4M
NHLBI NIH HHS R01 HL153988
6 · The paper itself

Abstract

Most people with Cystic Fibrosis (PwCF) harbor Cystic Fibrosis Transmembrane Conductance (CFTR) mutations that respond to highly effective CFTR modulators (HEM); however, a small fraction of non-responsive variants will require alternative approaches for treatment. Furthermore, the long-term goal to develop a cure for CF will require novel therapeutic strategies. Nucleic acid-based approaches offer the potential to address all CF-causing mutations and possibly a cure for all PwCF. In this minireview, we discuss current knowledge, recent progress, and critical questions surrounding the topic of Gene-, RNA-, and ASO-based therapies for the treatment of Cystic Fibrosis (CF).

Indexed as

Cystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorGenetic TherapyHumansMutationRNACystic Fibrosis Transmembrane Conductance RegulatorRNAASOCystic FibrosisDeliveryGeneRNATherapeutic

Identifiers

PMID36658041
PMCPMC10012168

What OpenQuestion holds

Textmetadata
LicenceTDM
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.