ReviewEMBO molecular medicine2013
Gene therapy on the move.
Review in EMBO molecular medicine, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 120 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
120 citing papers in PubMed.
- AAV8-mediated mouse/human PROC expression rescues thrombophilia in hereditary protein C-deficient mice.Gene therapy · 2026Article
- AAV Gene Therapy for MPS IVA with Induction of Immune Tolerance via Oral Administration of Epitope Peptides of N-Acetylgalactosamine-6-sulfate Sulfatase.International journal of molecular sciences · 2026Article
- Porphyrin-based MOFs for Gene Delivery in Cancer Therapy: Recent Advances and Progress.Current pharmaceutical design · 2026Review
- Advances in hydrogel-mediated gene therapy in ophthalmology: future directions and therapeutic potential.Regenerative biomaterials · 2026Review
- Cancer gene therapy: historical perspectives, current applications, and future directions.Functional & integrative genomics · 2025Review
- Longitudinal imaging of therapeutic enzyme expression after gene therapy for Fabry disease using positron emission tomography and the radiotracer [Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Gene therapy: principles, challenges and use in clinical practice.Wiener klinische Wochenschrift · 2025Review
- C3H10T1/2 Mesenchymal Stem Cell Line as a New In Vitro Tool for Studying Adipocyte Dedifferentiation.Biology · 2025Article
- [Research progresses in gene therapy for hepatolenticular degeneration].Zhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology · 2025Review
- Cell Therapies and Gene Therapy for Diabetes: Current Progress.Current diabetes reviews · 2025Review
- Immunometabolic crossroads: infections as bidirectional modulators in diabetes and metabolic syndromes.Frontiers in endocrinology · 2025Review
- Nanoemulsions Based Therapeutic Strategies: Enhancing Targeted Drug Delivery against Breast Cancer Cells.International journal of nanomedicine · 2025Review
- Hybrid Nanoparticles for Cancer Theranostics: A Critical Review on Design, Synthesis, and Multifunctional Capabilities.Current medicinal chemistry · 2025Review
- Revolutionizing medicine: Harnessing plant-derived vesicles for therapy and drug transport.Heliyon · 2024Review
- Navigating the intricate in-vivo journey of lipid nanoparticles tailored for the targeted delivery of RNA therapeutics: a quality-by-design approach.Journal of nanobiotechnology · 2024Review
- Targeted gene delivery systems for T-cell engineering.Cellular oncology (Dordrecht, Netherlands) · 2024Review
- Gene therapy for polygenic or complex diseases.Biomarker research · 2024Review
- Understanding the Mechanisms of Diminished Ovarian Reserve: Insights from Genetic Variants and Regulatory Factors.Reproductive sciences (Thousand Oaks, Calif.) · 2024Review
- Therapeutic potential of adiponectin in prediabetes: strategies, challenges, and future directions.Therapeutic advances in endocrinology and metabolism · 2024Review
- CRISPR-Cas9 in basic and translational aspects of cancer therapy.BioImpacts : BI · 2024Review
60 more citing papers are in PubMed but not listed here.
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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The first gene therapy clinical trials were initiated more than two decades ago. In the early days, gene therapy shared the fate of many experimental medicine approaches and was impeded by the occurrence of severe side effects in a few treated patients. The understanding of the molecular and cellular mechanisms leading to treatment- and/or vector-associated setbacks has resulted in the development of highly sophisticated gene transfer tools with improved safety and therapeutic efficacy. Employing these advanced tools, a series of Phase I/II trials were started in the past few years with excellent clinical results and no side effects reported so far. Moreover, highly efficient gene targeting strategies and site-directed gene editing technologies have been developed and applied clinically. With more than 1900 clinical trials to date, gene therapy has moved from a vision to clinical reality. This review focuses on the application of gene therapy for the correction of inherited diseases, the limitations and drawbacks encountered in some of the early clinical trials and the revival of gene therapy as a powerful treatment option for the correction of monogenic disorders.
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.