ArticleGene therapy2009
Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints.
Article in Gene therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 54 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
54 citing papers in PubMed, 72 citations in OpenAlex.
- Identification and Phylogenetic Characterisation of Novel Adeno-Associated Virus Capsids in Non-Human Primate Tissues.Cell proliferation · 2026Article
- CapBuild: a cloud-native tool for adeno-associated virus capsid engineering.Nucleic acids research · 2025Article
- Mapping administration route-dependent transduction profiles of commonly used AAV variants in mice by barcode amplicon sequencing.Molecular therapy. Methods & clinical development · 2025Article
- Adeno-associated viral tools to trace neural development and connectivity across amphibians.Developmental cell · 2025Article
- Recent Advances in Designing Adeno-Associated Virus-Based Vaccines Against Viral Infections.Pharmaceutics · 2024Review
- Machine-learning-guided Directed Evolution for AAV Capsid Engineering.Current pharmaceutical design · 2024Review
- Intravitreal injection of a rationally designed AAV capsid library in non-human primate identifies variants with enhanced retinal transduction and neutralizing antibody evasion.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Correlating physicochemical and biological properties to define critical quality attributes of a rAAV vaccine candidate.Molecular therapy. Methods & clinical development · 2023Article
- Determination of AAV properties by single amino acids: Go(o)d is in the details.Molecular therapy. Methods & clinical development · 2022Article
- Ancestral library identifies conserved reprogrammable liver motif on AAV capsid.Cell reports. Medicine · 2022Article
- Identification of Broadly Applicable Adeno-Associated Virus Vectors by Systematic Comparison of Commonly Used Capsid VariantsHuman gene therapy · 2022Article
- Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- Fantastic AAV Gene Therapy Vectors and How to Find Them-Random Diversification, Rational Design and Machine Learning.Pathogens (Basel, Switzerland) · 2022Review
- Serotype-dependent recombinant adeno-associated vector (AAV) infection of Epstein-Barr virus-positive B-cells, towards recombinant AAV-based therapy of focal EBV + lymphoproliferative disorders.Virology journal · 2021Article
- Frustration and Direct-Coupling Analyses to Predict Formation and Function of Adeno-Associated Virus.Biophysical journal · 2021Article
- Answered and Unanswered Questions in Early-Stage Viral Vector Transduction Biology and Innate Primary Cell Toxicity forFrontiers in immunology · 2021Review
- Novel AAV44.9-Based Vectors Display Exceptional Characteristics for Retinal Gene Therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- AAV Entry: Filling in the Blanks.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- Gene Therapy Leaves a Vicious Cycle.Frontiers in oncology · 2019Review
- A Novel Triple-Mutant AAV6 Capsid Induces Rapid and Potent Transgene Expression in the Muscle and Respiratory Tract of Mice.Molecular therapy. Methods & clinical development · 2018Article
Corrections and comments
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Authors and funding
10 authors at 3 institutions in 2 countries.
Funding
Abstract
Vectors based on the adeno-associated virus (AAV) are attractive and versatile vehicles for in vivo gene transfer. The virus capsid is the primary interface with the cell that defines many pharmacological, immunological and molecular properties. Determinants of these interactions are often restricted to a limited number of capsid amino acids. In this study, a portfolio of novel AAV vectors was developed after a structure-function analysis of naturally occurring AAV capsid isolates. Singletons, which are particular residues on the AAV capsid that were variable in otherwise conserved amino acid positions, were found to impact on vector's ability to be manufactured or to transduce. Data for those residues that mapped to monomer-monomer interface regions on the particle structure suggested a role in particle assembly. The change of singleton residues to the conserved amino acid resulted in the rescue of many isolates that were defective on initial isolation. This led to the development of an AAV vector portfolio that encompasses six different clades and 3 other distinct AAV niches. Evaluation of the in vivo gene transfer efficiency of this portfolio after intravenous and intramuscular administration highlighted a clade-specific tropism. These studies further the design and selection of AAV capsids for gene therapy applications.
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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.