DK2826860T3 - CNS targeting AAV vectors and methods for their use - Google Patents

CNS targeting AAV vectors and methods for their use Download PDF

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Publication number
DK2826860T3
DK2826860T3 DK14181861.7T DK14181861T DK2826860T3 DK 2826860 T3 DK2826860 T3 DK 2826860T3 DK 14181861 T DK14181861 T DK 14181861T DK 2826860 T3 DK2826860 T3 DK 2826860T3
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raav
cns
administration
mir
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DK14181861.7T
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Guangping Gao
Hongwei Zhang
Hongyan Wang
Zuoshang Xu
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Univ Massachusetts
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/86—Viral vectors
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    • C12N15/86—Viral vectors
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    • C12Y305/01015—Aspartoacylase (3.5.1.15)
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
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DK14181861.7T 2010-04-23 2011-04-22 CNS targeting AAV vectors and methods for their use DK2826860T3 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US32762710P 2010-04-23 2010-04-23
EP11772784.2A EP2561073B1 (en) 2010-04-23 2011-04-22 Cns targeting aav vectors and methods of use thereof

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DK14181861.7T DK2826860T3 (en) 2010-04-23 2011-04-22 CNS targeting AAV vectors and methods for their use
DK11772784.2T DK2561073T3 (en) 2010-04-23 2011-04-22 Aav vectors targeted to central nervous system and methods of use thereof

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US (8) US9102949B2 (enExample)
EP (6) EP3540055A1 (enExample)
JP (6) JP5963743B2 (enExample)
CA (3) CA2833908C (enExample)
DK (2) DK2826860T3 (enExample)
ES (2) ES2605305T3 (enExample)
WO (1) WO2011133890A1 (enExample)

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