Gene therapy for neurometabolic diseases

Defining, characterizing and applying gene therapy strategies for rare diseases affecting the nervous system, particularly those with neurometabolic involvement.

• Development of new treatments for megalencephalic leukoencephalopathy with subcortical cysts (MLC).  
• Gene therapy for Amyotrophic Lateral Sclerosis by targeting the correction of different molecular mechanisms. 
• New gene therapy strategies for Wolfram syndrome. 
• Study of neurodegeneration in the lysosomal disease Mucopolysaccharidosis type VII and its correction by gene therapy. 

Our research interests are focused on the development of strategies of gene therapy for diseases affecting both the central and peripheral nervous system and on the elucidation of the molecular mechanisms implicated in the development of these pathologies, combining the use of animal models, iPSC, and viral vectors. Our studies are designed to generate not only basic scientific data, but also at translational level, to provide patents, which value the research carried out in our University and attract the interest of biotechnological companies.