Abstract: The present invention refers to nucleotide sequences used for driving the expression of a therapeutic gene, preferably FVIII and/or its variants specifically in endothelial cells and/or hematopoietic, preferably myeloid cells. The sequences are useful for gene and/or cell therapy, preferably for treating hemophilia, more preferably type A hemophilia.

Abstract: The present invention refers to a method for inducing pluripotent stem cells starting from somatic cells isolated from healthy and/or diseased individuals. The diseased individual is preferably affected by a genetic disease such as type A hemophilia, and the somatic cells from the diseased individual are genetically corrected for the mutation causing the disease preferably after being reprogrammed by the method of the present invention. A further aspect of the present invention refers to a method for differentiating induced pluripotent stem cells or embryonic stem cell-like into endothelial cells. Moreover, the present invention refers to the use of these cells as a medicament for treating a disease, in particular, a genetic disease such as type A hemophilia.

Abstract: Sequences derived from Stabilin-2 promoter are used as a promoter to drive the expression of a gene specifically into endothelial cells, preferably said gene is a therapeutic gene, such as FVIII or any gene involved in the coagulation cascade. Moreover, the use of these sequences is for medical purposes, in particular, to treat a genetic disease, such as hemophilia, preferably by gene and/or cellular therapy.

Abstract: The present invention refers to nucleotide sequences used for driving the expression of a therapeutic gene, preferably FVIII and/or its variants specifically in endothelial cells and/or hematopoietic, preferably myeloid cells. The sequences are useful for gene and/or cell therapy, preferably for treating hemophilia, more preferably type A hemophilia.

Abstract: The present invention provides methods of treating hemophilia in a subject, the methods comprising transplanting a therapeutically effective amount of bone marrow, cord blood, cord blood or bone marrow fraction expressing Factor VIII, mononuclear cells, or mesenchymal stromal cells from a donor to the subject with hemophilia. The present invention also provides compositions for treating hemophilia.