ArticleJournal of translational medicine2019
A few ethical issues in translational research for gene and cell therapy.
Article in Journal of translational medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 1 of them a synthesis that pooled it.
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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.
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Who cites it
20 citing papers in PubMed, 1 synthesis or guideline pooled it, 43 citations in OpenAlex.
- Bioethics of somatic gene therapy: what do we know so far?Current medical research and opinion · 2023Pooled it
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- mRNA delivery systems 2.0: Engineering extrahepatic delivery for non-vaccine therapeutics.Materials today. Bio · 2025Review
- A Survey to Assess the Knowledge and Perceptions about Gene Therapy in Indian Haemophilia Patients.Indian journal of hematology & blood transfusion : an official journal of Indian Society of Hematology and Blood Transfusion · 2025Article
- The Pathophysiology of Psychiatric Disorders and the Therapeutic Potential of Mesenchymal Stem Cells.Psychiatry investigation · 2025Article
- Diverse potential of chimeric antigen receptor-engineered cell therapy: Beyond cancer.Clinical and translational medicine · 2025Review
- Advancing the Battle against Cystic Fibrosis: Stem Cell and Gene Therapy Insights.Current medical science · 2024Review
- Engineering signalling pathways in mammalian cells.Nature biomedical engineering · 2024Review
- The implications of waiving local clinical trials for drugs in India: a double-edged sword?The Lancet regional health. Southeast Asia · 2024Article
- Therapeutic Application and Structural Features of Adeno-Associated Virus Vector.Current issues in molecular biology · 2024Review
- Gene therapy-based strategies for spinal muscular atrophy-an Asia-Pacific perspective.Molecular and cellular pediatrics · 2023Review
- Review
- Medical student acceptance on gene therapy to increase children's well-being with genetic diseases: a study in Indonesia.Future science OA · 2022Article
- Ethical and practical considerations for cell and gene therapy toward an HIV cure: findings from a qualitative in-depth interview study in the United States.BMC medical ethics · 2022Article
- Gene Therapy Evidence Generation and Economic Analysis: Pragmatic Considerations to Facilitate Fit-for-Purpose Health Technology Assessment.Frontiers in public health · 2022Review
- Gene Therapy Targeting p53 and KRAS for Colorectal Cancer Treatment: A Myth or the Way Forward?International journal of molecular sciences · 2021Review
- Updating the ethical guidance for gene and cell therapy research participation.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Article
- Promises and challenges of adoptive T-cell therapies for solid tumours.British journal of cancer · 2021Review
- Genome Editing for Rare Diseases.Current stem cell reports · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundAlthough translational research for drug development can provide patients with valuable therapeutic resources it is not without risk, especially in the early-phase trials that present the highest degree of uncertainty. With the extraordinary evolution of biomedical technologies, a growing number of innovative products based on human cells and gene therapy are being tested and used as drugs. Their use on humans poses several challenges.
methodsIn this work, we discuss some ethical issues related to gene and cell therapies translational research. We focus on early-phase studies analysing the regulatory approach of Europe and the United States. We report the current recommendations and guidelines of international scientific societies and European and American regulatory authorities.
resultsThe peculiarity of human cell- or tissue-based products and gene therapy has required the development of specific regulatory tools that must be continually updated in line with the progress of the research. The ethics of translational research for these products also requires further considerations, particularly with respect to the specificity of the associated risk profiles.
conclusionsAn integrated ethical approach that aims for transparency and regulation of development processes, the support of independent judgment in clinical trials and the elimination of unregulated and uncontrolled grey areas of action are necessary to move gene and cell therapy forward.
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What 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.