Adeno-Associated Viral Vectors for the Gene Therapy of Metabolic Diseases
AssigneesUniversitat Autonoma De Barcelona, Ulf Per Gustav Smith
InventorsUlf Per Gustav SMITH, Fatima Maria BOSCH TUBERT, Ivet ELIAS PUIGDOMENECH
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Abstract
The present invention discloses adeno-associated viral vectors useful in gene therapy methods for the treatment of obesity, insulin resistance, type 2 diabetes, liver cirrhosis and non-alcoholic fatty liver disease (NAFLD)/non-alcoholic steatohepatitis (NASH). The invention also relates to polynucleotides, plasmids, vectors and methods for the production of such adeno-associated viral vectors. The invention also relates to pharmaceutical compositions comprising said vectors.
Claims21
1-68. (canceled)
An adeno-associated viral vector comprising a recombinant viral genome wherein said recombinant viral genome comprises an expression cassette comprising a transcriptional regulatory region operatively linked to a nucleotide sequence encoding BMP4 or a functionally equivalent variant thereof with the proviso that when the expression cassette comprises a chicken β-actin promoter and cytomegalovirus enhancer, the Kozak sequence GCCACCATGG (SEQ ID NO:
- 1), a nucleotide sequence encoding rat BMP4, a woodchuck hepatitis B virus post-regulatory element (WPRE) and a bovine growth hormone polyadenylation signal, and said expression cassette is flanked by AAV2 ITRs, then the serotype of the adeno-associated viral vector is not AAV2.
A polynucleotide comprising an expression cassette flanked by adeno-associated virus ITRs wherein said expression cassette comprises a transcriptional regulatory region operatively linked to a nucleotide sequence encoding BMP4 or a functionally equivalent variant thereof with the proviso that said expression cassette is not an expression cassette comprising a chicken β-actin promoter and cytomegalovirus enhancer, the Kozak sequence GCCACCATGG (SEQ ID NO:
- 1), a nucleotide sequence encoding rat BMP4, a woodchuck hepatitis B virus post-regulatory element (WPRE) and a bovine growth hormone polyadenylation signal.
Dependencies are inferred from the claim text.