ReviewPharmaceuticals (Basel, Switzerland)2023
Current State of Human Gene Therapy: Approved Products and Vectors.
Review in Pharmaceuticals (Basel, Switzerland), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 51 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
51 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Antisense Versus Antigene in the Computer-Aided Design of Triplex-Forming Oligonucleotides (TFO): Insights from a Dual-Method Review, Combining Bibliometric and Systematic Review.International journal of molecular sciences · 2025Pooled it
- Base editing rescues a hereditary motor neuron disease in mouse and patient-derived iPSC organoid models.Molecular therapy. Advances · 2026Article
- Optimal Hypoxia Mimetic Small Molecules for Enhancing Angiogenic Properties of Stem Cells From Human Exfoliated Deciduous Teeth.International endodontic journal · 2026Article
- Angiogenic Doping: Plausible Yet Difficult to Detect.Sports medicine (Auckland, N.Z.) · 2026Review
- Epigenetic regulation of transgenes.Journal of biotechnology · 2026Review
- Anion-Exchange Membrane Chromatography for the Separation of Empty and Full Adeno-Associated Viral Capsids.Biotechnology journal · 2026Article
- Applications of Recombinant DNA Technology in Medicine: A Comprehensive Review.Molecular biotechnology · 2026Review
- IDR-driven LLPS of GAS2L3 scaffolds CHMP4B condensates to accelerate cytokinesis in hepatocellular carcinoma cells.Journal of advanced research · 2026Article
- Dual targeted gene delivery strategy mediated by GalNAc-modified lipid nanoparticles enhances liver regeneration through specific knockdown of MKK4.Materials today. Bio · 2026Article
- Tailoring virus-inspired nanoparticles for advanced drug and gene delivery.Materials today. Bio · 2026Review
- Breaking the Barrier of Brain Disease Therapeutics: Advocating Targeted Drug Delivery for Improved Neuro-Resident Interventions.AAPS PharmSciTech · 2026Review
- Therapeutic microRNAs: Mechanisms, Delivery, and Clinical Translation in Oncology.International journal of molecular sciences · 2026Review
- Promises past and future - Gene therapy and the actualisation of future expectations.Social science & medicine (1982) · 2026Article
- Organoids and organs-on-chips for accelerating R&D and clinical translation in Orthopaedics: Emerging opportunities and regulatory pathways.Journal of orthopaedic translation · 2026Review
- Landscape ofMolecular therapy. Methods & clinical development · 2025Article
- Gene Therapy for Wet Age-Related Macular Degeneration.Bioengineering (Basel, Switzerland) · 2025Review
- Cell and gene therapy in Germany: three decades of advanced technologies.Immunotherapy · 2025Article
- Beyond single targets: leveraging degeneracy in sodium channels for osteoarthritis analgesia.Pain reports · 2025Article
- Exploring the Use of Viral Vectors Pseudotyped with Viral Glycoproteins as Tools to Study Antibody-Mediated Neutralizing Activity.Microorganisms · 2025Review
- Synthetic G-quadruplex components for predictable, precise two-level control of mammalian recombinant protein expression.Nucleic acids research · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In the realm of gene therapy, a pivotal moment arrived with Paul Berg's groundbreaking identification of the first recombinant DNA in 1972. This achievement set the stage for future breakthroughs. Conditions once considered undefeatable, like melanoma, pancreatic cancer, and a host of other ailments, are now being addressed at their root cause-the genetic level. Presently, the gene therapy landscape stands adorned with 22 approved in vivo and ex vivo products, including IMLYGIC, LUXTURNA, Zolgensma, Spinraza, Patisiran, and many more. In this comprehensive exploration, we delve into a rich assortment of 16 drugs, from siRNA, miRNA, and CRISPR/Cas9 to DNA aptamers and TRAIL/APO2L, as well as 46 carriers, from AAV, AdV, LNPs, and exosomes to naked mRNA, sonoporation, and magnetofection. The article also discusses the advantages and disadvantages of each product and vector type, as well as the current challenges faced in the practical use of gene therapy and its future potential.
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.