ReviewSignal transduction and targeted therapy2024
Adeno-associated virus as a delivery vector for gene therapy of human diseases.
Review in Signal transduction and targeted therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 538 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
538 citing papers in PubMed, 1 synthesis or guideline pooled it, 601 citations in OpenAlex.
- Incidence, timing, and clinical significance of adverse immune events after gene replacement therapy: A systematic review and meta-analysis.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Pooled it
- Pharmacodynamics, Efficacy, and Safety of Intraputaminal Eladocagene Exuparvovec Administered to Pediatric Patients With Aromatic L-Amino Acid Decarboxylase Deficiency Using an MR-Compatible Cannula: 48 Weeks of Follow-Up.Journal of inherited metabolic disease · 2026Trial
- Review
- Clinical immunogenicity in rAAV gene therapy: Insights and implications.Molecular therapy. Advances · 2026Review
- Benchmarking the next generation of brain-penetrant AAV capsids.Molecular therapy. Advances · 2026Article
- RNA-encoded PGT121-LS anti-HIV antibody: Comprehensive preclinical characterization and translational pharmacokinetics.Molecular therapy. Nucleic acids · 2026Article
- In vivo immune cell engineering from bench to clinical reality.Pharmaceutical science advances · 2026Review
- Purification of Advanced Therapeutics by Flow-Through Pseudo-Affinity Chromatography: Bi-Specific mABs, Fc Fusion Proteins, and Adeno-Associated Viruses.Biotechnology and bioengineering · 2026Article
- Global epidemiology of myocarditis: disparities in age, sex and geography.Nature reviews. Cardiology · 2026Review
- AAV-mediated delivery of leptin but not adiponectin improves metabolic health in a pre-clinical mouse model of congenital generalised lipodystrophy.Gene therapy · 2026Article
- Engineered human Argonaute 2 for targeted C-to-U RNA base editing.Nature biotechnology · 2026Article
- Prevalence of adeno-associated virus type 3B neutralizing antibodies in cohorts of healthy donors and Wilson's disease adults.Gene therapy · 2026Article
- Cells heal themselves: gene-edited hematopoietic stem cells restore frataxin deficiency in multiple organs.The Journal of clinical investigation · 2026Article
- Intrinsic Fluorescence as a Probe of Structural Dynamics and Interactions of Human Adenovirus with Surfactants and Liposomes.Journal of fluorescence · 2026Article
- mRNA Therapeutics for Skin Rejuvenation: From Aging Atlases to Clinical Translation.Experimental dermatology · 2026Review
- Patient-derived teratomas as a humanized platform for dystrophin restoration by ex vivo cell therapy and in vivo base editing.Experimental & molecular medicine · 2026Article
- Glomerular epithelial protein-1 promotes reparative dentin formation in vivo.Journal of molecular histology · 2026Article
- Subcellular photochemistry for precision spatial protein targeting.Nature reviews. Chemistry · 2026Review
- Targeted AAV gene therapy for neuroblastoma via direct capsid-antibody coupling.EMBO molecular medicine · 2026Article
- Controllable gene delivery via masked adeno-associated viral vectors.Nature materials · 2026Article
478 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 at 3 institutions in 2 countries.
Funding
Abstract
Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different tissues. Recombinant AAV (rAAV) has been engineered for enhanced specificity and developed as a tool for treating various diseases. However, as rAAV is being more widely used as a therapy, the increased demand has created challenges for the existing manufacturing methods. Seven rAAV-based gene therapy products have received regulatory approval, but there continue to be concerns about safely using high-dose viral therapies in humans, including immune responses and adverse effects such as genotoxicity, hepatotoxicity, thrombotic microangiopathy, and neurotoxicity. In this review, we explore AAV biology with an emphasis on current vector engineering strategies and manufacturing technologies. We discuss how rAAVs are being employed in ongoing clinical trials for ocular, neurological, metabolic, hematological, neuromuscular, and cardiovascular diseases as well as cancers. We outline immune responses triggered by rAAV, address associated side effects, and discuss strategies to mitigate these reactions. We hope that discussing recent advancements and current challenges in the field will be a helpful guide for researchers and clinicians navigating the ever-evolving landscape of rAAV-based gene therapy.
Indexed as
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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.