ReviewDiscovery medicine
State-of-the-art human gene therapy: part I. Gene delivery technologies.
Review in Discovery medicine. 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, 85 citations in OpenAlex.
- AAV2-Retro-Mediated Gene Transfer Selectively Targets Outer Retinal Cells Following Intravitreal Injection.Investigative ophthalmology & visual science · 2026Article
- Gene therapy: principles, challenges and use in clinical practice.Wiener klinische Wochenschrift · 2025Review
- Gene Therapy in Rare Genetic Disorders: Current Progress and Future Perspectives.Current genomics · 2025Review
- Evaluation of a rapid multi-attribute combinatorial high-throughput UV-Vis/DLS/SLS analytical platform for rAAV quantification and characterization.Molecular therapy. Methods & clinical development · 2024Article
- Delivery of DNA-Based Therapeutics for Treatment of Chronic Diseases.Pharmaceutics · 2024Review
- Adeno-associated virus as a delivery vector for gene therapy of human diseases.Signal transduction and targeted therapy · 2024Review
- Current Landscape of Gene Therapy for the Treatment of Cardiovascular Disorders.Current gene therapy · 2024Review
- Engineering and Bio/Nanotechnological Applications of Virus Particles.Sub-cellular biochemistry · 2024Review
- Sustained and Efficient Delivery of Antivascular Endothelial Growth Factor by the Adeno-associated Virus for the Treatment of Corneal Neovascularization: An Outlook for Its Clinical Translation.Journal of ophthalmology · 2024Review
- Gene Therapy for Inherited Hearing Loss: Updates and Remaining Challenges.Audiology research · 2023Review
- Gene Therapy for Regenerative Medicine.Pharmaceutics · 2023Review
- Nanoparticle Mediated Gene Therapy: A Trailblazer Armament to Fight CNS Disorders.Current medicinal chemistry · 2023Review
- Update on Viral Gene Therapy Clinical Trials for Retinal Diseases.Human gene therapy · 2022Review
- Gene therapy in neuromuscular disorders.Arquivos de neuro-psiquiatria · 2022Review
- [Precision medicine in pediatric neurology exemplified by the new treatment forms].Der Nervenarzt · 2022Review
- Design and Delivery of SINEUP: A New Modular Tool to Increase Protein Translation.Methods in molecular biology (Clifton, N.J.) · 2022Article
- Efficacious, safe, and stable inhibition of corneal neovascularization by AAV-vectored anti-VEGF therapeutics.Molecular therapy. Methods & clinical development · 2021Article
- Vector Strategies to Actualize B Cell-Based Gene Therapies.Journal of immunology (Baltimore, Md. : 1950) · 2021Review
- Non-viral gene delivery of HIF-1α promotes angiogenesis in human adipose-derived stem cells.Acta biomaterialia · 2020Article
- Lysosomal storage diseases: current therapies and future alternatives.Journal of molecular medicine (Berlin, Germany) · 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 at 1 institution in 1 country.
Funding
Abstract
Safe and effective gene delivery is a prerequisite for successful gene therapy. In the early age of human gene therapy, setbacks due to problematic gene delivery vehicles plagued the exciting therapeutic outcome. However, gene delivery technologies rapidly evolved ever since. With the advancement of gene delivery techniques, gene therapy clinical trials surged during the past decade. As the first gene therapy product (Glybera) has obtained regulatory approval and reached clinic, human gene therapy finally realized the promise that genes can be medicines. The diverse gene delivery techniques available today have laid the foundation for gene therapy applications in treating a wide range of human diseases. Some of the most urgent unmet medical needs, such as cancer and pandemic infectious diseases, have been tackled by gene therapy strategies with promising results. Furthermore, combining gene transfer with other breakthroughs in biomedical research and novel biotechnologies opened new avenues for gene therapy. Such innovative therapeutic strategies are unthinkable until now, and are expected to be revolutionary. In part I of this review, we introduced recent development of non-viral and viral gene delivery technology platforms. As cell-based gene therapy blossomed, we also summarized the diverse types of cells and vectors employed in ex vivo gene transfer. Finally, challenges in current gene delivery technologies for human use were discussed.
Indexed as
Identifiers
25091489PMC4440413W2278507501What 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.