Evidence map›Paper›PMID 39172342›Full record

ArticleMedicine, health care, and philosophy2024

What is a cure through gene therapy? An analysis and evaluation of the use of "cure".

Lieke Baas, Karina Meijer, Annelien L Bredenoord, Rieke van der Graaf

Abstract read
In one paragraph

Article in Medicine, health care, and philosophy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing 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

8 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
  4. Normalization in hemophilia: conceptual foundations and clinical implications.Research and practice in thrombosis and haemostasis · 2025
    Review
  5. Review
  6. Article
  7. Can hemophilia be cured? It depends on the definition.Research and practice in thrombosis and haemostasis · 2024
    Review
  8. 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

4 authors.

Lieke BaasDepartment of Bioethics and Health Humanities, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands. L.baas-3@umcutrecht.nl.ORCID http://orcid.org/0000-0001-6032-2712
Karina MeijerDepartment of Hematology, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands.
Annelien L BredenoordErasmus School of Philosophy, Erasmus University Rotterdam, Rotterdam, the Netherlands.
Rieke van der GraafDepartment of Bioethics and Health Humanities, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands.

Funding

Nederlandse Organisatie voor Wetenschappelijk Onderzoek NWA.1160.18.038
6 · The paper itself

Abstract

The development of gene therapy has always come with the expectation that it will offer a cure for various disorders, of which hemophilia is a paradigm example. However, although the term is used regularly, it is unclear what exactly is meant with "cure". Therefore, the aim of this paper is to analyse how the concept of cure is used in practice and evaluate which of the interpretations is most suitable in discussions surrounding gene therapy. We analysed how cure is used in four different medical fields where the concept raises discussion. We show that cure can be used in three different ways: cure as normalization of the body, cure as obtaining a normal life, or cure as a change in identity. We argue that since cure is a practical term, its interpretation should be context-specific and the various uses can exist simultaneously, as long as their use is suitable to the function the notion of cure plays in each of the settings. We end by highlighting three different settings in the domain of hemophilia gene therapy in which the term cure is used and explore the function(s) it serves in each setting. We conclude that in the clinical application of gene therapy, it could be better to abandon the term cure, whereas more modest and specified definitions of cure are required in the context of health resource allocation decisions and decisions on research funding.

Indexed as

Genetic TherapyHemophilia AHumansPhilosophy, MedicalCureDiseaseEthicsGene therapyHealthPhilosophy of medicine

Identifiers

PMID39172342
PMCPMC11519194

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.