AAV/UPR-plus virus, UPR-plus fusion protein, genetic treatment method and its use in treatment of neurodegenerative diseases, such as Parkinson's disease and Huntington's disease, among others
First Claim
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1. An adeno-associated vector (AAV), comprising a recombinant viral genome wherein said genome comprises an expression cassette comprising a transcription regulatory region specific for neuronal tissues operatively bound to a UPRplus polynucleotide of interest coding a fusion protein.
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Abstract
The present invention presents a sequence of the AAV/UPR-plus virus, a genetic treatment method and its use in the treatment of neurodegenerative diseases, such as Parkinson'"'"'s and Huntington'"'"'s diseases, among others, as presented in the in vitro studies shown in FIG. 14/17.
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81 Claims
- 1. An adeno-associated vector (AAV), comprising a recombinant viral genome wherein said genome comprises an expression cassette comprising a transcription regulatory region specific for neuronal tissues operatively bound to a UPRplus polynucleotide of interest coding a fusion protein.
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- 42. A polynucleotide, comprising an expression cassette flanked by the ITRs of an adeno-associated virus, where said expression cassette comprises a promoter, a coding region for immune response, and a polynucleotide of interest that encodes a UPRplus fusion protein.
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69. A plasmid, comprising sequences of an adeno-associated virus, an expression cassette flanked by ITRs of the adeno-associated virus, wherein said expression cassette comprises a promoter, a coding region for immune response and a polynucleotide of interest, wherein the plasmid is deposited in the international body of biological deposits, Instituto de Investigaciones Agropecuarias de Chile, INIA, under a deposit number selected from the group consisting of RGM 2231, RGM 2232, RGM 2233, RGM 2234, RGM 2235 and RGM 2236.
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Specification