ReviewMolecular therapy : the journal of the American Society of Gene Therapy2009
Accepting risk in clinical research: is the gene therapy field becoming too risk-averse?
Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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
24 citing papers in PubMed.
- Trial
- Advancements in Gene Delivery using Nucleic Acid Loaded Nanoparticles for Region Specific Delivery in Alzheimer's Disease.Molecular neurobiology · 2026Review
- A conceptual model and practical guidance for the development, administration, and evaluation of individualized therapies.Frontiers in medicine · 2025Article
- Public perceptions and willingness to accept somatic gene therapy: A Belgian survey study.Open research Europe · 2025Article
- Cutting-edge Strategies for Overcoming Therapeutic Barriers in Alzheimer's Disease.Current pharmaceutical design · 2025Review
- The acceptability of the risk of death in the treatment of respiratory diseases in France.Health economics review · 2024Review
- Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care.Biotech (Basel (Switzerland)) · 2024Review
- Article
- Ethical challenges for a new generation of early-phase pediatric gene therapy trials.Genetics in medicine : official journal of the American College of Medical Genetics · 2021Review
- Gene Therapy Knowledge and Attitude Among Healthcare Professionals: A Cross-Sectional Study.Frontiers in public health · 2021Article
- Voretigene Neparvovec and Gene Therapy for Leber's Congenital Amaurosis: Review of Evidence to Date.The application of clinical genetics · 2020Review
- Genome editing: A perspective on the application of CRISPR/Cas9 to study human diseases (Review).International journal of molecular medicine · 2019Review
- Regulatory and Scientific Advancements in Gene Therapy: State-of-the-Art of Clinical Applications and of the Supporting European Regulatory Framework.Frontiers in medicine · 2017Review
- Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.Human gene therapy · 2014Review
- Office based muscle biopsy using Vacora vacuum assisted biopsy system.African journal of medicine and medical sciences · 2012Article
- When ethics constrains clinical research: trial design of control arms in "greater than minimal risk" pediatric trials.Human gene therapy · 2011Article
- A DNA microarray-based analysis of the host response to a nonviral gene carrier: a strategy for improving the immune response.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- Genome-wide target profiling of piggyBac and Tol2 in HEK 293: pros and cons for gene discovery and gene therapy.BMC biotechnology · 2011Article
- A transposon and transposase system for human application.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Review
- Gene therapy in Alzheimer's disease - potential for disease modification.Journal of cellular and molecular medicine · 2010Review
Corrections and comments
- Commented on by
- Commented on by
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Risk is an inescapable aspect of clinical research and is increasingly pertinent to the gene therapy field as the imperative for clinical trial activity grows. In recent years, the widely reported occurrence of serious adverse events (SAEs) in gene therapy studies, including trials for ornithine transcarbamylase (OTC) deficiency, X-linked severe combined immunodeficiency (SCID-X1), and rheumatoid arthritis, has heightened fear in public perceptions of gene therapy. Although it is essential to be cognizant of the risks involved in gene therapy research, there is a danger that gene therapy may become too risk-averse. If the field is to make progress, it is necessary to understand how risk is defined in gene therapy research, how understandings of risk differ, how risk is assessed, how decisions about risk are made, and how gene therapy risks are communicated to subjects and research participants during the informed consent process. In addition to minimizing the risks of clinical research through extensive preclinical safety studies, attention should be given to how decisions about risk and risk acceptability are made by researchers and subjects, and to the methods used to communicate risks to patients. Critical attention to risk will help ensure that the safety of subjects is protected, while also enabling research to develop better treatments for patients.
Indexed as
Identifiers
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.