  | Immune tolerance induction through enforced expression of autoantigens in hematopoietic cells using gene therapy and non myeloablative strategies (in collaboration with Drs. C. Espejo, H. Eixarch & X. Montalbán, Clinical Neuroimmunology Unit / CEM-Cat).
PI: Jordi Barquinero Mañez
|
| Applications of induced pluripotent stem cells (iPSC) on the diagnosis and therapy of diseases of the hematopoietic system (in collaboration with research groups at CIEMAT, CMRB & Hospital Niño Jesús). PI: Juan Bueren (CIEMAT, Madrid) |
| Preclinical development of a gene therapy strategy in hematopoietic cells for mitochondrial neurogastrointestinal encephalopathy (MNGIE) (in collaboration with Drs. R. Martí, J. Torres & A. Andreu, mitochondrial and neuromuscular pathology group).
PI: Ramón Martí Seves
|
| Use of patient-specific induced pluripotent stem cells to improve diagnosis and treatment of hemophilia A
PI: Jordi Barquinero Mañez, Lluis Martorell Cedres, Rafael Parra López
|
|
Patient-specific iPSCs offer novel ways to model human diseases in a way that captures the patients’ genomic complexity. Specifically, this project is aimed to use and optimize the current protocols for in vitro differentiation of iPSCs into functional hepatocytes and endothelial cells (EC); to generate hemophilia A (HA) patient-specific iPSCs lines from selected patients, which will be used for assessing the effectiveness of drugs that promote nonsense codon readthrough for treating HA; and optimizing genetic correction of HA patient-specific iPSCs or iPSCs-derived cells.
|
|
