UPO book

Brief Research Description

1. Cell and gene therapy approaches for the treatment of haemophilia A. Identification and study of cells mainly involved in the production of coagulation factor VIII with the aim of using them for cell therapy or as targets for gene therapy of haemophilia A.

2. Study of the involvement of myeloid cells in the immune response to FVIII, both during replacement therapy and following gene therapy.

3. Approaches to treat and prevent hemarthrosis and avoid its progression into osteoarthritis.

4. Transplantation of different liver cells into the liver and their engraftment. Study of the engraftment in the liver of different cells of liver origin after transplantation with the aim of defining the best conditions to be used in cell and gene therapy protocols for liver regeneration and/or the treatment of metabolic diseases.

Keywords

Cell therapy, Gene therapy, Hemophilia A, Liver regeneration, Hematopoietic cells 

PubMed