ReviewMolecular therapy : the journal of the American Society of Gene Therapy2020
Variability in Genome Editing Outcomes: Challenges for Research Reproducibility and Clinical Safety.
Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 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
28 citing papers in PubMed, 42 citations in OpenAlex.
- Targeted Nanoparticle Delivery CRISPR/Cas9: overcoming biological barriers, enhancing stability, and improving therapeutic precision.International journal of pharmaceutics: X · 2026Review
- TrxR Inhibition and Nrf2-FOXO3 Modulation by Repurposed Drugs: A Redox Strategy to Reverse Cancer Multidrug Resistance.Drug development research · 2026Review
- The future of pediatric gene therapy: CRISPR-Cas9, AI, and personalized medicine.Pediatric research · 2026Review
- Measurement and clinical interpretation of CRISPR off-targets.Nature genetics · 2026Review
- CRISPR/Cas9 in cancer therapy: clinical translation, mechanistic strategies, and therapeutic directions.Frontiers in oncology · 2026Review
- Clinical perspective: Advancing hemophilia treatment through gene therapy approaches.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Applications of Gene Editing and Nanotechnology in Stem Cell-Based Therapies for Human Diseases.Stem cell reviews and reports · 2025Review
- High throughput functional profiling of genes at intraocular pressure loci reveals distinct networks for glaucoma.Human molecular genetics · 2024Article
- A Fluorescent Reporter Mouse forThe CRISPR journal · 2023Article
- Precision RNA base editing with engineered and endogenous effectors.Nature biotechnology · 2023Review
- Hidden prevalence of deletion-inversion bi-alleles in CRISPR-mediated deletions of tandemly arrayed genes in plants.Nature communications · 2023Article
- Correspondence of Yolk Sac and Embryonic Genotypes in F0 Mouse CRISPants.Medical research archives · 2023Article
- CRISPR-Cas9 base editors and their current role in human therapeutics.Cytotherapy · 2023Review
- Production of Cloned Pigs by Handmade Cloning.Methods in molecular biology (Clifton, N.J.) · 2023Article
- Gold Nanoparticle-Mediated Gene Therapy.Cancers · 2022Review
- CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy.Stem cells translational medicine · 2022Article
- Addressing the dark matter of gene therapy: technical and ethical barriers to clinical application.Human genetics · 2022Review
- CRISPRi for specific inhibition of miRNA clusters and miRNAs with high sequence homology.Scientific reports · 2022Article
- Prospects of Non-Coding Elements in Genomic DNA Based Gene Therapy.Current gene therapy · 2022Review
- DAJIN enables multiplex genotyping to simultaneously validate intended and unintended target genome editing outcomes.PLoS biology · 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
5 authors at 3 institutions in 2 countries.
Funding
Abstract
Genome editing tools have already revolutionized biomedical research and are also expected to have an important impact in the clinic. However, their extensive use in research has revealed much unpredictability, both off and on target, in the outcome of their application. We discuss the challenges associated with this unpredictability, both for research and in the clinic. For the former, an extensive validation of the model is essential. For the latter, potential unpredicted activity does not preclude the use of these tools but requires that molecular evidence to underpin the relevant risk:benefit evaluation is available. Safe and successful clinical application will also depend on the mode of delivery and the cellular context.
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.