Our research focuses on developing gene therapy approaches for highly prevalent diseases, such as diabetes mellitus and obesity, and rare metabolic/neurodegenerative diseases. Obesity and type 2 diabetes, strongly associated with deficits in cognitive function, are a major health problem because of their alarming growing prevalence worldwide. Novel adeno-associated viral (AAV) vector-mediated gene therapy strategies are used to target these pathophysiological processes. We are also developing gene therapies for Mucopolysaccharidosis, a group of devastating lysosomal storage diseases that currently lack effective treatments. We aim at developing gene therapies that are minimally invasive, safe and could guarantee maximal efficacy in the most affected organs. The know-how derived from these studies is also used to assess the effectiveness of AAV-based gene therapeutics products to counteract other neurodegenerative diseases and rare diseases affecting the skeletal system in mouse models.

Fàtima Bosch Tubert