ArticleCurrent gene therapy2017
First-in-human Phase 1 CRISPR Gene Editing Cancer Trials: Are We Ready?
Article in Current gene therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 44 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
44 citing papers in PubMed.
- Delivering the future of immunotherapy: A state-of-the-art review of gene editing in immune cells with lipid nanoparticles.Materials today. Bio · 2026Review
- CRISPR-Edited Cell Lines: A New Era in Functional Oncology Research.Current pharmaceutical design · 2026Review
- CRISPR-engineered microbiome: living therapeutics revolutionize blood cancer immunotherapy.NPJ biofilms and microbiomes · 2025Review
- Nanocarrier-Based Systems for Targeted Delivery: Current Challenges and Future Directions.MedComm · 2025Review
- Designer Organs: Ethical Genetic Modifications in the Era of Machine Perfusion.Annual review of biomedical engineering · 2025Review
- CRISPR/Cas9 gene editing: a novel strategy for fighting drug resistance in respiratory disorders.Cell communication and signaling : CCS · 2024Review
- Review
- An Update on the Application of CRISPR Technology in Clinical Practice.Molecular biotechnology · 2024Review
- CRISPR-Cas9 Gene Editing: Curing Genetic Diseases by Inherited Epigenetic Modifications.Global medical genetics · 2024Review
- Site-specific transgene integration in chimeric antigen receptor (CAR) T cell therapies.Biomarker research · 2023Review
- Initial heritable genome editing: mapping a responsible pathway from basic research to the clinic.Medicine, health care, and philosophy · 2023Article
- Integration of CRISPR/Cas9 with artificial intelligence for improved cancer therapeutics.Journal of translational medicine · 2022Review
- Chemistry of Peptide-Oligonucleotide Conjugates: A Review.Molecules (Basel, Switzerland) · 2021Review
- CRISPR/Cas9 revitalizes adoptive T-cell therapy for cancer immunotherapy.Journal of experimental & clinical cancer research : CR · 2021Review
- New Insights into the Therapeutic Applications of CRISPR/Cas9 Genome Editing in Breast Cancer.Genes · 2021Review
- CRISPR-Cas9: A Preclinical and Clinical Perspective for the Treatment of Human Diseases.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- CRISPR/Cas System and Factors Affecting Its Precision and Efficiency.Frontiers in cell and developmental biology · 2021Review
- CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside.British journal of haematology · 2021Review
- Toward Anticipatory Governance of Human Genome Editing: A Critical Review of Scholarly Governance Discourse.Journal of responsible innovation · 2021Article
- Harnessing the potential of CRISPR-based platforms to advance the field of hospital medicine.Expert review of anti-infective therapy · 2020Review
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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
A prospective first-in-human Phase 1 CRISPR gene editing trial in the United States for patients with melanoma, synovial sarcoma, and multiple myeloma offers hope that gene editing tools may usefully treat human disease. An overarching ethical challenge with first-in-human Phase 1 clinical trials, however, is knowing when it is ethically acceptable to initiate such trials on the basis of safety and efficacy data obtained from pre-clinical studies. If the pre-clinical studies that inform trial design are themselves poorly designed - as a result of which the quality of pre-clinical evidence is deficient - then the ethical requirement of scientific validity for clinical research may not be satisfied. In turn, this could mean that the Phase 1 clinical trial will be unsafe and that trial participants will be exposed to risk for no potential benefit. To assist sponsors, researchers, clinical investigators and reviewers in deciding when it is ethically acceptable to initiate first-in-human Phase 1 CRISPR gene editing clinical trials, structured processes have been developed to assess and minimize translational distance between pre-clinical and clinical research. These processes draw attention to various features of internal validity, construct validity, and external validity. As well, the credibility of supporting evidence is to be critically assessed with particular attention to optimism bias, financial conflicts of interest and publication bias. We critically examine the pre-clinical evidence used to justify the first-inhuman Phase 1 CRISPR gene editing cancer trial in the United States using these tools. We conclude that the proposed trial cannot satisfy the ethical requirement of scientific validity because the supporting pre-clinical evidence used to inform trial design is deficient.
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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.