ReviewGenetics in medicine : official journal of the American College of Medical Genetics2021
Ethical challenges for a new generation of early-phase pediatric gene therapy trials.
Review in Genetics in medicine : official journal of the American College of Medical Genetics, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 1 of them a synthesis that pooled 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.
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.
Who cites it
9 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Who should be included in first-in-human trials? A systematic review of reasons.Journal of translational medicine · 2025Pooled it
- Expertise of European Clinical Trial Units in Conducting and Managing Cross-Border Pediatric Clinical Trials for Rare Diseases.Therapeutic innovation & regulatory science · 2026Article
- Guidance on communication and informed consent with patients and their families for experimental individualized treatments.American journal of human genetics · 2026Review
- The next phase in Long COVID research: addressing the ethical challenges in trials of disease-modifying treatments.EClinicalMedicine · 2026Review
- Role of Pharmacy Professionals in Gene Therapy Based on Adeno-Associated Viruses: Treatment of Hemophilia as a Template of Care.The Canadian journal of hospital pharmacy · 2025Review
- A conceptual model and practical guidance for the development, administration, and evaluation of individualized therapies.Frontiers in medicine · 2025Article
- Medical decision-making competence regarding puberty suppression: perceptions of transgender adolescents, their parents and clinicians.European child & adolescent psychiatry · 2023Article
- Familial Hypertrophic Cardiomyopathy: Diagnosis and Management.Vascular health and risk management · 2023Review
- Investigating attitudes toward prenatal diagnosis and fetal therapy for spinal muscular atrophy.Prenatal diagnosis · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors.
Funding
Abstract
After decades of setbacks, gene therapy (GT) is experiencing major breakthroughs. Five GTs have received US regulatory approval since 2017, and over 900 others are currently in development. Many of these GTs target rare pediatric diseases that are severely life-limiting, given a lack of effective treatments. As these GTs enter early-phase clinical trials, specific ethical challenges remain unresolved in three domains: evaluating risks and potential benefits, selecting participants fairly, and engaging with patient communities. Drawing on our experience as clinical investigators, basic scientists, and bioethicists involved in a first-in-human GT trial for an ultrarare pediatric disease, we analyze these ethical challenges and offer points to consider for future GT trials.
Indexed as
Identifiers
34234300What OpenQuestion holds
Registered trials
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.