A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.
Brain. 2018.
PMID: 29788236
Free PMC article.
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A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.
Brain. 2018 Jul 1;141(7):2014-2031. doi: 10.1093/brain/awy126.
Brain. 2018.
PMID: 29788236
Free PMC article.
To overcome these hurdles, a variety of capsid engineering methods have been utilized to generate capsids with improved transduction properties. Here we describe an alternative approach to capsid engineering, which draws on the natural evolution of the vir …
To overcome these hurdles, a variety of capsid engineering methods have been utilized to generate capsids with improved transd …
Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties.
Nat Commun. 2020 Jun 30;11(1):3279. doi: 10.1038/s41467-020-17047-1.
Nat Commun. 2020.
PMID: 32606306
Free PMC article.
Recombinant adeno-associated viruses (rAAVs) are currently considered the safest and most reliable gene delivery vehicles for human gene therapy. ...Here, we describe a novel capsid identified in a human clinical sample by high-throughput, long-read se …
Recombinant adeno-associated viruses (rAAVs) are currently considered the safest and most reliable gene delivery vehicles for …
New AAV9 engineered variants with enhanced neurotropism and reduced liver off-targeting in mice and marmosets.
iScience. 2024 Apr 18;27(5):109777. doi: 10.1016/j.isci.2024.109777. eCollection 2024 May 17.
iScience. 2024.
PMID: 38711458
Free PMC article.
Although adeno-associated virus 9 (AAV9) has been highly exploited as delivery platform for gene-based therapies, its efficacy is hampered by low efficiency in crossing the adult blood-brain barrier (BBB) and pronounced targeting to the liver upon intravenous …
Although adeno-associated virus 9 (AAV9) has been highly exploited as delivery platform for gene-based therapies, its e …
A combination of mutations enhances the neurotropism of AAV-2.
Virology. 2005 Oct 25;341(2):203-14. doi: 10.1016/j.virol.2005.06.051. Epub 2005 Aug 15.
Virology. 2005.
PMID: 16102794
Free article.
Our objective was to generate innocuous gene vehicles that would be effectively taken up by axons and then home to the neuron cell bodies. Vectors derived from Adeno-Associated Virus (AAV), a harmless human parvovirus, offer strong starting candidates for der …
Our objective was to generate innocuous gene vehicles that would be effectively taken up by axons and then home to the neuron cell bodies. V …
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