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    Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10

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    Authors
    Yang, Bin
    Li, Shaoyong
    Wang, Hongyan
    Guo, Yansu
    Gessler, Dominic J.
    Cao, Chunyan
    Su, Qin
    Kramer, Joshua
    Zhong, Li
    Ahmed, Seemin Seher
    Zhang, Hongwei
    He, Ran
    Desrosiers, Ronald C.
    Brown, Robert H. Jr.
    Xu, Zuoshang
    Gao, Guangping
    Show allShow less
    Student Authors
    Seemin Seher Ahmed
    UMass Chan Affiliations
    Department of Neurology
    Department of Microbiology and Physiological Systems
    Gene Therapy Center
    Department of Biochemistry and Molecular Pharmacology
    Document Type
    Journal Article
    Publication Date
    2014-07-01
    Keywords
    Animals; Brain; Callithrix; Central Nervous System; Dependovirus; Male; Mice; MicroRNAs; Primates
    Genetics
    Genetics and Genomics
    Molecular Genetics
    Nervous System Diseases
    Therapeutics
    Virology
    
    Metadata
    Show full item record
    Link to Full Text
    http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4089005/
    Abstract
    Some recombinant adeno-associated viruses (rAAVs) can cross the neonatal blood-brain barrier (BBB) and efficiently transduce cells of the central nervous system (CNS). However, in the adult CNS, transduction levels by systemically delivered rAAVs are significantly reduced, limiting their potential for CNS gene therapy. Here, we characterized 12 different rAAVEGFPs in the adult mouse CNS following intravenous delivery. We show that the capability of crossing the adult BBB and achieving widespread CNS transduction is a common character of AAV serotypes tested. Of note, rAAVrh.8 is the leading vector for robust global transduction of glial and neuronal cell types in regions of clinical importance such as cortex, caudate-putamen, hippocampus, corpus callosum, and substantia nigra. It also displays reduced peripheral tissue tropism compared to other leading vectors. Additionally, we evaluated rAAVrh.10 with and without microRNA (miRNA)-regulated expressional detargeting from peripheral tissues for systemic gene delivery to the CNS in marmosets. Our results indicate that rAAVrh.8, along with rh.10 and 9, hold the best promise for developing novel therapeutic strategies to treat neurological diseases in the adult patient population. Additionally, systemically delivered rAAVrh.10 can transduce the CNS efficiently, and its transgene expression can be limited in the periphery by endogenous miRNAs in adult marmosets.
    Source
    Mol Ther. 2014 Jul;22(7):1299-309. doi: 10.1038/mt.2014.68. Epub 2014 Apr 30. Link to article on publisher's site
    DOI
    10.1038/mt.2014.68
    Permanent Link to this Item
    http://hdl.handle.net/20.500.14038/33352
    PubMed ID
    24781136
    Related Resources
    Link to Article in PubMed
    ae974a485f413a2113503eed53cd6c53
    10.1038/mt.2014.68
    Scopus Count
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    Morningside Graduate School of Biomedical Sciences Scholarly Publications

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