Evidence map›Paper›PMID 37470194›Full record

ReviewJournal of inherited metabolic disease2024

Fetal gene therapy.

Simon N Waddington, William H Peranteau, Ahad A Rahim, Ashley K Boyle, Manju A Kurian, Paul Gissen, Jerry K Y Chan, Anna L David

Abstract readReview
In one paragraph

Review in Journal of inherited metabolic disease, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing 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

11 citing papers in PubMed.

  1. Considerations for early life genetic therapies in cystic fibrosis.American journal of physiology. Lung cellular and molecular physiology · 2026
    Review
  2. Defining a Midgestational Window forbioRxiv : the preprint server for biology · 2026
    Article
  3. Medizinische Genetik : Mitteilungsblatt des Berufsverbandes Medizinische Genetik e.V · 2026
    Article
  4. Article
  5. Review
  6. Article
  7. ABI and generative biology: A new paradigm for gene therapy, genome engineering, and engineered cell therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  8. Article
  9. Article
  10. Review
  11. 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

8 authors.

Simon N WaddingtonEGA Institute for Women's Health, University College London, London, UK.ORCID 0000-0003-4970-4730
William H PeranteauThe Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.ORCID 0000-0003-1608-861X
Ahad A RahimUCL School of Pharmacy, University College London, London, UK.ORCID 0000-0003-0044-0949
Ashley K BoyleEGA Institute for Women's Health, University College London, London, UK.
Manju A KurianDevelopmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, GOS-Institute of Child Health, University College London, London, UK.ORCID 0000-0003-3529-5075
Paul GissenGreat Ormond Street Institute of Child Health, University College London, London, UK.
Jerry K Y ChanDepartment of Reproductive Medicine, KK Women's and Children's Hospital, Singapore, Singapore.
Anna L DavidEGA Institute for Women's Health, University College London, London, UK.ORCID 0000-0002-0199-6140

Funding

In utero gene editing to cure a metabolic liver diseaseR01DK123049 · NIDDK · CHILDREN'S HOSP OF PHILADELPHIA · PI PERANTEAU, WILLIAM H. · 2020 to 2024
$3.7M
In utero CRISPR-mediated gene editing to cure congenital monogenic disordersDP2HL152427 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI PERANTEAU, WILLIAM H. · 2019 to 2019
$2.6M
Prenatal pulmonary cell gene editing to cure monogenic lung diseasesR01HL151352 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI PERANTEAU, WILLIAM H. · 2020 to 2022
$1.2M
Medical Research Council MR/N019075/1Medical Research Council MR/N026101/1Medical Research Council MR/R015325/1Medical Research Council MR/R025134/1Medical Research Council MR/S009434/1Medical Research Council MR/S036784/1Medical Research Council MR/T016809/1Medical Research Council MR/T044853/1NHLBI NIH HHS DP2 HL152427NHLBI NIH HHS R01 HL151352NIDDK NIH HHS R01 DK123049
6 · The paper itself

Abstract

Fetal gene therapy was first proposed toward the end of the 1990s when the field of gene therapy was, to quote the Gartner hype cycle, at its "peak of inflated expectations." Gene therapy was still an immature field but over the ensuing decade, it matured and is now a clinical and market reality. The trajectory of treatment for several genetic diseases is toward earlier intervention. The ability, capacity, and the will to diagnose genetic disease early-in utero-improves day by day. A confluence of clinical trials now signposts a trajectory toward fetal gene therapy. In this review, we recount the history of fetal gene therapy in the context of the broader field, discuss advances in fetal surgery and diagnosis, and explore the full ambit of preclinical gene therapy for inherited metabolic disease.

Indexed as

Fetal TherapiesGenetic TherapyFemaleHumansPregnancygene therapyinherited metabolic disease

Identifiers

PMID37470194
PMCPMC10799196

What OpenQuestion holds

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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.