ReviewGene therapy2009
Tailoring the AAV vector capsid for gene therapy.
Review in Gene therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 91 papers, 2 of them syntheses 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
91 citing papers in PubMed, 2 syntheses or guidelines pooled it, 159 citations in OpenAlex.
- Gene Therapy Advances: A Meta-Analysis of AAV Usage in Clinical Settings.Frontiers in medicine · 2021Pooled it
- Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays.International journal of molecular sciences · 2020Pooled it
- USP24-dependent STAT2 stabilization mediates physiologic and pathologic bone formation.Cell death & disease · 2026Article
- Screening of Single-Domain Antibodies to Adeno-Associated Viruses with Cross-Serotype Specificity and a Wide pH Tolerance.Viruses · 2025Article
- Generation and Treatment of a Novel Severe Model of Visceral Gaucher Disease by Genetic Therapy.Pharmaceutics · 2025Article
- Review
- A RANKL-UCHL1-sCD13 negative feedback loop limits osteoclastogenesis in subchondral bone to prevent osteoarthritis progression.Nature communications · 2024Article
- PETA: evaluating the impact of protein transfer learning with sub-word tokenization on downstream applications.Journal of cheminformatics · 2024Article
- New AAV9 engineered variants with enhanced neurotropism and reduced liver off-targeting in mice and marmosets.iScience · 2024Article
- AAV-based gene editing of type 1 collagen mutation to treat osteogenesis imperfecta.Molecular therapy. Nucleic acids · 2024Article
- Recent developments in gene therapy research in India.Journal of biosciences · 2024Review
- Durable contraception in the female domestic cat using viral-vectored delivery of a feline anti-Müllerian hormone transgene.Nature communications · 2023Article
- Reversed Phase-Liquid Chromatography for Recombinant AAV Genome Integrity Assessment.Analytical chemistry · 2023Article
- Schnurri-3 inhibition suppresses bone and joint damage in models of rheumatoid arthritis.Proceedings of the National Academy of Sciences of the United States of America · 2023Article
- AAV-mediated delivery of osteoblast/osteoclast-regulating miRNAs for osteoporosis therapy.Molecular therapy. Nucleic acids · 2022Article
- Endothelial Retargeting of AAV9 In Vivo.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2022Article
- Viral vector-based gene therapies in the clinic.Bioengineering & translational medicine · 2022Review
- UniDecCD: Deconvolution of Charge Detection-Mass Spectrometry Data.Analytical chemistry · 2021Article
- A Native Mass Spectrometry-Based Assay for Rapid Assessment of the Empty:Full Capsid Ratio in Adeno-Associated Virus Gene Therapy Products.Analytical chemistry · 2021Article
- An AAV-based, room-temperature-stable, single-dose COVID-19 vaccine provides durable immunogenicity and protection in non-human primates.Cell host & microbe · 2021Article
31 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
3 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
A number of preclinical studies have shown the adeno-associated virus (AAV) to be an efficient vehicle for gene therapy. Clinical studies successfully demonstrated its potential for in vivo gene transfer. The complexity of host-vector interactions when progressing from small to large animal models, and eventually to humans, has impeded translation of AAV technology to the clinic. One approach to address this complexity has been to explore the biological characteristics of variations in AAV capsid structure. Initial strategies characterized the naturally occurring capsid variants from mammalian species. The structural and functional knowledge gathered on these natural AAV variants as vectors has led to the first series of second-generation vectors that aim at specifically improving certain properties by rational design of the capsid. A third exciting approach uses directed evolution to isolate vectors that are able to overcome selective pressures applied in the laboratory and thereby steer the capsid to evolve toward improved functionality.
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.