ReviewFrontiers in medicine2017
Regulatory and Scientific Advancements in Gene Therapy: State-of-the-Art of Clinical Applications and of the Supporting European Regulatory Framework.
Review in Frontiers in medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers, 1 of them a synthesis that pooled it.
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
23 citing papers in PubMed, 1 synthesis or guideline pooled it, 56 citations in OpenAlex.
- ATMP Environmental Exposure Assessment in European Healthcare Settings: A Systematic Review of the Literature.Frontiers in medicine · 2021Pooled it
- Advanced therapy medicinal products in Saudi Arabia: Regulation and development.Regenerative therapy · 2026Review
- Pathophysiological mechanisms of organ injury in Fabry disease: Update via multi-omics.Genes & diseases · 2026Review
- Recent Advances in the Non-viral Delivery of Genes to Central Nervous System Disorders.Cellular and molecular neurobiology · 2026Review
- Unconventional bioprinting modalities for advanced tissue biofabrication.Biomaterials · 2026Review
- Why advanced therapy medicinal products struggle in clinical translation: an in-depth analysis of developmental challenges in the EU.Frontiers in medicine · 2026Review
- Bioprinted Constructs in the Regulatory Landscape: Current State and Future Perspectives.Advanced materials (Deerfield Beach, Fla.) · 2026Review
- Non-viral approaches in CAR-NK cell engineering: connecting natural killer cell biology and gene delivery.Journal of nanobiotechnology · 2024Review
- Gene Therapy with Chitosan Nanoparticles: Modern Formulation Strategies for Enhancing Cancer Cell Transfection.Pharmaceutics · 2024Review
- Navigating Gene Therapy Access: The Case of Bulgaria in the Context of the EU Regulatory Landscape.Healthcare (Basel, Switzerland) · 2024Article
- Regulatory Framework, Challenges, and Initial Strategic Planning for Advanced Therapy Products (PTAs) Development in Brazil.Therapeutic innovation & regulatory science · 2024Review
- Adenoviral vectors for cardiovascular gene therapy applications: a clinical and industry perspective.Journal of molecular medicine (Berlin, Germany) · 2022Review
- Neurosurgery at the crossroads of immunology and nanotechnology. New reality in the COVID-19 pandemic.Advanced drug delivery reviews · 2022Review
- Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs.Molecular therapy. Methods & clinical development · 2021Review
- α-Galactosidase A Augmentation by Non-Viral Gene Therapy: Evaluation in Fabry Disease Mice.Pharmaceutics · 2021Article
- Oxidative Stress and New Pathogenetic Mechanisms in Endothelial Dysfunction: Potential Diagnostic Biomarkers and Therapeutic Targets.Journal of clinical medicine · 2020Review
- Delivery across the blood-brain barrier: nanomedicine for glioblastoma multiforme.Drug delivery and translational research · 2020Review
- Nanomedicines to Deliver mRNA: State of the Art and Future Perspectives.Nanomaterials (Basel, Switzerland) · 2020Review
- Development and Use of Gene Therapy Orphan Drugs-Ethical Needs for a Broader Cooperation Between the Pharmaceutical Industry and Society.Frontiers in medicine · 2020Article
- EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation.Molecular therapy. Methods & clinical development · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Advanced therapy medicinal products (ATMPs) have a massive potential to address existing unmet medical needs. Specifically, gene therapy medicinal products (GTMPs) may potentially provide cure for several genetic diseases. In Europe, the ATMP regulation was fully implemented in 2009 and, at this point, the Committee for Advanced Therapies was created as a dedicated group of specialists to evaluate medicinal products requiring specific expertise in this area. To date, there are three authorized GTMPs, and the first one was approved in 2012. Broad research has been conducted in this field over the last few decades and different clinical applications are being investigated worldwide, using different strategies that range from direct gene replacement or addition to more complex pathways such as specific gene editing or RNA targeting. Important safety risks, limited efficacy, manufacturing hurdles, or ethical conflicts may represent challenges in the success of a candidate GTMP. During the development process, it is fundamental to take such aspects into account and establish overcoming strategies. This article reviews the current European legal framework of ATMPs, provides an overview of the clinical applications for approved and investigational GTMPs, and discusses critical challenges in the development of GTMPs.
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.