Virus AAV/IGF2, genetic treatment method and use thereof in protein misfolding-related diseases, such as Huntington's disease
AssigneeUniversidad De Chile
InventorsClaudio Andrés Hetz Flores, Paula García Huerta
Life of this patent
Estimating the term…
Abstract
The invention relates to the expression of two molecules in viral vectors AAV/IGF2-HA and AAV/IGF2, the associated method and use thereof in the amelioration of protein misfolding-related diseases, such as Huntington's disease, as presented in the in vivo models in FIGS. 11/19.
Claims9
A therapeutic method for treatment of Huntington's disease in a subject in need thereof, the method comprising:
- administering a therapeutically effective amount of a pharmaceutical composition to said subject directly to striatum in brain by stereotactic intrastriatal injection, wherein said pharmaceutical composition comprises:
- an adeno-associated virus (AAV), serotype AAV2, comprising a recombinant viral genome, wherein said genome comprises an expression cassette comprising a CAG promoter operably linked to a polynucleotide of interest that encodes for human IGF2;
- and a pharmaceutically acceptable excipient.
A plasmid, comprising sequences of an adeno-associated vector, and an expression cassette flanked by ITRs of an adeno-associated virus,
- wherein said expression cassette comprises a promoter, and a polynucleotide of interest that encodes IGF2,
- wherein the plasmid is one of those deposited at an international agent of biological deposit, Chilean Collection of Microbial Genetic Resources (CChRGM), having deposit numbers RGM2335 and RGM2336.
A pharmaceutical composition for treatment of Huntington's disease in a subject in need thereof, the pharmaceutical composition comprising:
- an adeno-associated virus (AAV), serotype AAV2, comprising a recombinant viral genome, wherein said genome comprises an expression cassette comprising a CAG promoter operably linked to a polynucleotide of interest that encodes for human IGF2, wherein said expression cassette is included in one of those plasmid deposited at international agent of biological deposit, Chilean Collection of Microbial Genetic Resources (CChRGM), having deposit numbers RGM2335 and RGM2336;
- and
- a pharmaceutically acceptable excipient,
- wherein the composition is formulated to be administered directly to brain of said subject.
Dependencies are inferred from the claim text.