Abstract: Described herein is a genetically modified plant or non-human animal having reduced expression of an endogenous target gene.

Abstract: Described herein is a gene construct comprising a nucleotide sequence encoding a fibroblast growth factor 21 (FGF21), for use in the treatment and/or prevention of a central nervous system (CNS) disorder or disease, or a condition associated therewith.

Abstract: The present invention relates to adeno-associated viral vector useful for transducing adipose tissue. The invention also relates to polynucleotides, plasmids, vectors and methods for the production of such adeno-associated viral vector. The invention also relates to gene therapy methods useful for the treatment of a disease that requires the regulation of the expression levels of a gene.

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.

Abstract: Described herein is a gene construct comprising a nucleotide sequence encoding a fibroblast growth factor 21 (FGF21), for use in the treatment and/or prevention of a metabolic disorder, wherein the therapy involves expression of the gene construct in the central nervous system (CNS).

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.

Abstract: The invention relates to a viral expression construct and related viral vector and composition and to their use wherein the construct and vector comprise elements a) and b): a) a nucleotide sequence encoding an insulin operably linked to a first promoter, b) a nucleotide sequence encoding a glucokinase operably linked to a second promoter and the viral expression construct and related viral vector comprise at least one of elements c), d) and e): c) the first and the second promoters are positioned in reverse orientation within the expression construct, d) the first and the second promoters are positioned in reverse orientation within the expression construct and are located adjacent to each other and e) the first promoter is a CMV promoter, preferably a mini CMV promoter.

Abstract: The present invention relates to adeno-associated viral vector useful for transducing adipose tissue. The invention also relates to polynucleotides, plasmids, vectors and methods for the production of such adeno-associated viral vector. The invention also relates to gene therapy methods useful for the treatment of a disease that requires the regulation of the expression levels of a gene.

Abstract: The present invention relates to adeno-associated viral vector useful for transducing adipose tissue. The invention also relates to polynucleotides, plasmids, vectors and methods for the production of such adeno-associated viral vector. The invention also relates to gene therapy methods useful for the treatment of a disease that requires the regulation of the expression levels of a gene.

Abstract: The invention relates to a viral expression construct and related viral vector and nucleic acid molecule and composition and to their use wherein said construct and vector are suitable for expression in a mammal and comprise a nucleotide sequence encoding a Fibroblast growth factor 21 (FGF21) to be expressed in liver, adipose tissue and/or skeletal muscle.

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.

Abstract: The present invention relates to adeno-associated viral vector useful for transducing adipose tissue. The invention also relates to polynucleotides, plasmids, vectors and methods for the production of such adeno-associated viral vector. The invention also relates to gene therapy methods useful for the treatment of a disease that requires the regulation of the expression levels of a gene.