Viral vectors for the treatment of diabetes
OwnerCristina Mallol Dominguez, Fatima Bosch Tubert +1inventors
InventorsCristina Mallol Dominguez, Fatima Bosch Tubert, Veronica Jimenez Cenzano
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Abstract
They are provided gene constructs comprising a nucleotide sequence encoding the Insulin-like growth factor 1 (IGF-1) of a mammal; and target sequences of a microRNA of a tissue where the expression of IGF-1 is wanted to be prevented, wherein the sequences (a) and (b) are operationally linked to a promoter of ubiquitous expression. Also provided are expression vectors comprising the gene construct and pharmaceutical compositions comprising them. They are useful in the treatment and/or prevention of diabetes mellitus in mammals, wherein a dysfunction and/or a loss of the beta-cells of the islets of Langerhans is present.
Claims1
An adeno-associated viral expression vector comprising a gene construct comprising:
- (a) a nucleotide sequence encoding the Insulin-like growth factor 1 (IGF-1) of a mammal;
- and
- (b) at least one target sequence of the microRNA-122a and at least one target sequence of the microRNA-1,
- wherein the sequences (a) and (b) are operationally linked to a ubiquitous promoter,
- wherein the vector comprises a sequence selected from SEQ ID NO:
- 30 and SEQ ID NO:
- 31.
Dependencies are inferred from the claim text.