The Cell Technology division is focused on the development of gene and cell therapy strategies for the treatment of inherited metabolic diseases. The lines of research include both the study of the basic biology of these diseases, as well as the development of cell and animal models based on genetic engineering techniques, such as the application of gene editing in diseases like Type I Hyperoxaluria or Pyruvate Deficiency. Kinase (PKD).
On the other hand, in the field of gene therapy, the Cell Technology division is focused on the development of lentiviral vectors for the treatment of Pyruvate Kinase Deficiency (PKD) and for the treatment of Congenital Dyserythropoietic Anemia type II (CDAII). These investigations have led to the development of a clinical trial for the treatment of patients with Pyruvate Kinase Deficiency.
For more information, consult the Cellular Technology division website.