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  5. A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency
 
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A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency
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A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.pdf (2.56 MB)
Published version
Author(s)
Tordo, Julie
O'Leary, Claire
Antunes, Andre SLM
Palomar, Nuria
Aldrin-Kirk, Patrick
more
Type
Journal Article
Abstract
Recombinant adeno-associated viruses (AAVs) are popular in vivo gene transfer vehicles. However, vector doses needed to achieve therapeutic effect are high and some target tissues in the central nervous system remain difficult to transduce. Gene therapy trials using AAV for the treatment of neurological disorders have seldom led to demonstrated clinical efficacy. Important contributing factors are low transduction rates and inefficient distribution of the vector. 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 virus and aims to yield capsids that are better suited to infect human tissues. We generated an AAV capsid to include amino acids that are conserved among natural AAV2 isolates and tested its biodistribution properties in mice and rats. Intriguingly, this novel variant, AAV-TT, demonstrates strong neurotropism in rodents and displays significantly improved distribution throughout the central nervous system as compared to AAV2. Additionally, sub-retinal injections in mice revealed markedly enhanced transduction of photoreceptor cells when compared to AAV2. Importantly, AAV-TT exceeds the distribution abilities of benchmark neurotropic serotypes AAV9 and AAVrh10 in the central nervous system of mice, and is the only virus, when administered at low dose, that is able to correct the neurological phenotype in a mouse model of mucopolysaccharidosis IIIC, a transmembrane enzyme lysosomal storage disease, which requires delivery to every cell for biochemical correction. These data represent unprecedented correction of a lysosomal transmembrane enzyme deficiency in mice and suggest that AAV-TT-based gene therapies may be suitable for treatment of human neurological diseases such as mucopolysaccharidosis IIIC, which is characterized by global neuropathology.
Date Issued
2018-07
Date Acceptance
2018-03-21
Citation
Brain, 2018, 141 (7), pp.2014-2031
URI
https://hdl.handle.net/10044/1/117061
URL
https://doi.org/10.1093/brain/awy126
DOI
https://www.dx.doi.org/10.1093/brain/awy126
ISSN
0006-8950
Publisher
Oxford University Pres
Start Page
2014
End Page
2031
Journal / Book Title
Brain
Volume
141
Issue
7
Copyright Statement
© The Author(s) (2018). Published by Oxford University Press on behalf of the Guarantors of Brain. This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0/), which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly cited. For commercial re-use, please contact journals.permissions@oup.com
License URL
http://creativecommons.org/licenses/by-nc/4.0/
Identifier
https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000438288100020&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=a2bf6146997ec60c407a63945d4e92bb
PII: 4996997
Subjects
adeno-associated virus
capsid engineering
Clinical Neurology
CONGENITAL AMAUROSIS
GENE-THERAPY VECTOR
HEPARAN-SULFATE PROTEOGLYCAN
HIPPOCAMPAL-LESIONS
Life Sciences & Biomedicine
lysosomal transmembrane enzyme
MOUSE MODEL
mucopolysaccharidosis
MUCOPOLYSACCHARIDOSIS-IIIB
NEURONAL CEROID-LIPOFUSCINOSIS
Neurosciences
Neurosciences & Neurology
neurotropism
RECEPTOR FOOTPRINT
SANFILIPPO-SYNDROME
Science & Technology
VIRAL VECTORS
Publication Status
Published
Coverage Spatial
England
Date Publish Online
2018-05-16
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