Research graph
References from A single amino acid variant in the variable region I of AAV capsid confers liver detargeting. Local targets link to admitted publications; unresolved targets remain external evidence.
Parvoviridae: the viruses and their replication.
2001 · External reference
Adeno-associated virus vector as a platform for gene therapy delivery
10.1038/s41573-019-0012-9 · 2019 · External reference
Adeno-associated viruses undergo substantial evolution in primates during natural infections
10.1073/pnas.0937739100 · 2003 · External reference
Clades of Adeno-associated viruses are widely disseminated in human tissues
10.1128/jvi.78.12.6381-6388.2004 · 2004 · External reference
Unresolved reference
External reference
Engineering adeno-associated virus vectors for gene therapy
10.1038/s41576-019-0205-4 · 2020 · External reference
AAV capsid structure and cell interactions
10.1007/978-1-61779-370-7_3 · 2011 · External reference
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector
10.1038/mt.2011.237 · 2012 · External reference
Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles
10.1128/jvi.00675-12 · 2012 · External reference
In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses
10.1128/jvi.00254-08 · 2008 · External reference
Structure of AAV-DJ, a retargeted gene therapy vector: cryo-electron microscopy at 4.5 A resolution
10.1016/j.str.2012.05.004 · 2012 · External reference
Mapping the AAV capsid host antibody response toward the development of second generation gene delivery vectors
10.3389/fimmu.2014.00009 · 2014 · External reference
Adeno-Associated Virus (AAV) gene delivery: dissecting molecular interactions upon cell entry
10.3390/v13071336 · 2021 · External reference
Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle
10.1038/nbt.1599 · 2010 · External reference
Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties
10.1038/s41467-020-17047-1 · 2020 · External reference
Two-year outcomes of valoctocogene roxaparvovec therapy for Hemophilia A
10.1056/nejmoa2211075 · 2023 · External reference
Gene therapy with etranacogene dezaparvovec for Hemophilia B
10.1056/nejmoa2211644 · 2023 · External reference
Alpha(1)-Antitrypsin deficiency
10.1056/nejmra1910234 · 2020 · External reference
Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques
10.1016/j.omtm.2024.101200 · 2024 · External reference
Unresolved reference
External reference
Ferret models of alpha-1 antitrypsin deficiency develop lung and liver disease
2022 · External reference
Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids
10.1016/j.omtm.2021.04.010 · 2021 · External reference
Ancestral library identifies conserved reprogrammable liver motif on AAV capsid
10.1016/j.xcrm.2022.100803 · 2022 · External reference
Unresolved reference
2015 · External reference
Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy
10.1016/j.jhep.2020.11.001 · 2021 · External reference
Liver injury in cynomolgus monkeys following intravenous and intrathecal scAAV9 gene therapy delivery
10.1016/j.ymthe.2023.07.020 · 2023 · External reference
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury
10.1016/j.ymthe.2024.02.002 · 2024 · External reference
An overview of nonclinical and clinical liver toxicity associated with AAV gene therapy
10.1177/01926233231201408 · 2023 · External reference
High-Throughput quantification of in vivo adeno-associated virus transduction with barcoded non-coding RNAs
10.1089/hum.2018.253 · 2019 · External reference
Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants
10.1038/s41467-020-19230-w · 2020 · External reference
Adeno-associated virus capsid proteins may play a role in transcription and second-strand synthesis of recombinant genomes
10.1128/jvi.02093-13 · 2014 · External reference
The AAV capsid can influence the epigenetic marking of rAAV delivered episomal genomes in a species dependent manner
10.1038/s41467-023-38106-3 · 2023 · External reference
Capsid-mediated control of adeno-associated viral transcription determines host range
10.1016/j.celrep.2024.113902 · 2024 · External reference
Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species
10.1016/j.cell.2021.08.028 · 2021 · External reference
An essential receptor for adeno-associated virus infection
10.1038/nature16465 · 2016 · External reference
Adeno-associated virus 2 bound to its cellular receptor AAVR
10.1038/s41564-018-0356-7 · 2019 · External reference
Structure of the gene therapy vector, adeno-associated virus with its cell receptor, AAVR
10.7554/elife.44707 · 2019 · External reference
Deamidation of amino acids on the surface of adeno-associated virus capsids leads to charge heterogeneity and altered vector function
10.1016/j.ymthe.2018.09.013 · 2018 · External reference
Redirecting AAV vectors to extrahepatic tissues
10.1016/j.ymthe.2023.10.005 · 2023 · External reference
Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
10.1038/nbt.3440 · 2016 · External reference
Highly conserved brain vascular receptor ALPL mediates transport of engineered AAV vectors across the blood-brain barrier
2025 · External reference
An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery
10.1126/science.adm8386 · 2024 · External reference
Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle
10.1089/hum.2010.003 · 2010 · External reference
A scalable and accurate method for quantifying vector genomes of recombinant adeno-associated viruses in crude lysate
10.1089/hgtb.2016.173 · 2017 · External reference
Adeno-Associated virus neutralizing antibodies in large animals and their impact on brain intraparenchymal gene transfer
10.1016/j.omtm.2018.09.003 · 2018 · External reference
The Galaxy platform for accessible, reproducible, and collaborative data analyses: 2024 update
10.1093/nar/gkae410 · 2024 · External reference
