AAV/UPR-plus virus, UPR-plus fusion protein, genetic treatment method and its use in the treatment of neurodegenerative diseases, such as Parkinson's disease and Huntington's disease, among others
AssigneeUniversidad De Chile
InventorsClaudio Hetz Flores, Rene Vidal Gomez
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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.
Claims18
An adeno-associated vector (AAV), comprising a recombinant viral genome wherein said genome comprises an expression cassette comprising a transcription regulatory region including a promoter specific for neuronal tissues operatively bound to a polynucleotide of interest coding a fusion protein, wherein the fusion protein comprises XBP1s, ATF6f, and a bridge or linker sequence.
A polynucleotide, comprising an expression cassette flanked by the ITRs of an adeno-associated virus, where said expression cassette comprises a promoter, and a polynucleotide of interest that encodes a fusion protein, wherein the fusion protein comprises XBP1s, ATF6f, and a bridge or linker sequence.
A plasmid 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.
Dependencies are inferred from the claim text.