Evidence map›Paper›PMID 39236265›Full record

ReviewAmerican journal of respiratory and critical care medicine2024

Lentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-in-Human Trial.

Jane C Davies, Deepika Polineni, A Christopher Boyd, Scott Donaldson, Deborah R Gill, Uta Griesenbach, Stephen C Hyde, Raksha Jain, Gerry McLachlan, Marcus A Mall and 1 more

Abstract readReview
In one paragraph

Review in American journal of respiratory and critical care medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.

0numbers the graph read from it
0cells of the map it votes in
26citing 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

26 citing papers in PubMed.

  1. Molecular therapy. Nucleic acids · 2026
    Article
  2. Review
  3. Review
  4. Lentiviral-mediated gene complementation to rescue pathogenic ABCA3 variants.American journal of respiratory cell and molecular biology · 2026
    Article
  5. Review
  6. Nucleic acid-based therapies for people with cystic fibrosis.The Cochrane database of systematic reviews · 2026
    Article
  7. Review
  8. Review
  9. Review
  10. Article
  11. Review
  12. Considerations for early life genetic therapies in cystic fibrosis.American journal of physiology. Lung cellular and molecular physiology · 2026
    Review
  13. Optimized AAV capsids robustly transduce airway epithelial cells.bioRxiv : the preprint server for biology · 2026
    Article
  14. Review
  15. Article
  16. Genetic medicines for epilepsy: unlocking new avenues for seizure control.Frontiers in bioengineering and biotechnology · 2026
    Review
  17. Review
  18. PRDM16 Reduces Cellular Senescence by Upregulating GSTM1.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025
    Article
  19. Review
  20. Precise progerin targeting using RfxCas13d: A therapeutic avenue for Hutchinson-Gilford progeria syndrome.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

11 authors.

Jane C DaviesNational Heart and Lung Institute, Imperial College London, London, United Kingdom.ORCID 0000-0003-3506-1199
Deepika PolineniDivision of Allergy and Pulmonary Medicine, Department of Pediatrics, Washington University School of Medicine in St. Louis, St. Louis, Missouri.ORCID 0000-0001-8551-3734
A Christopher BoydU.K. Respiratory Gene Therapy Consortium, Oxford, United Kingdom.
Scott DonaldsonDivision of Pulmonary Diseases and Critical Care Medicine, Department of Medicine, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.ORCID 0000-0003-4802-0388
Deborah R GillU.K. Respiratory Gene Therapy Consortium, Oxford, United Kingdom.ORCID 0000-0002-5264-054X
Uta GriesenbachNational Heart and Lung Institute, Imperial College London, London, United Kingdom.ORCID 0000-0002-8254-4540
Stephen C HydeU.K. Respiratory Gene Therapy Consortium, Oxford, United Kingdom.ORCID 0000-0002-8877-4005
Raksha JainDepartment of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, Texas.ORCID 0000-0002-2407-7374
Gerry McLachlanU.K. Respiratory Gene Therapy Consortium, Oxford, United Kingdom.ORCID 0000-0001-5362-9804
Marcus A MallDepartment of Pediatric Respiratory Medicine, Immunology, and Critical Care Medicine, Charité - Universitätsmedizin Berlin, Berlin, Germany.ORCID 0000-0002-4057-2199
Eric W F W AltonNational Heart and Lung Institute, Imperial College London, London, United Kingdom.ORCID 0000-0003-3852-6764

Funding

Boehringer IngelheimWellcome Trust
6 · The paper itself

Abstract

Cystic fibrosis (CF) is a genetic disease caused by mutations in the

Indexed as

Cystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorGenetic TherapyGenetic VectorsLentivirusHumansMutationCFTR protein, humanCystic Fibrosis Transmembrane Conductance RegulatorCFTRgenetic therapyintegrating vectorslentivirusmutation-agnostic treatment

Identifiers

PMID39236265
PMCPMC11716034

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.