Gene Therapy Research
Our laboratory focuses on developing gene therapies for fatal neurological diseases through design of AAV vectors carrying therapeutic genes and engineering new AAV capsids with enhanced in vivo gene delivery to central nervous system and other target tissues.
Miguel Sena-Esteves
Principal Investigator
Leading the team, Miguel is an accomplished Investigator focused in Gene Therapy, holding a PhD in biomedical sciences.
Tay-Sachs & Sandhoff
Among all of the lysosomal storage disorders, the GM2 gangliosidoses are the most investigated subcategory caused by the impaired degradation.
GM1-Gangliosidosis
An autosomal recession disease caused by mutations in the GLB1 gene encoding for the lysosomal acid beta-galactosidase (Bgal) enzyme.
Huntington's Disease
The most common among those movement diseases collectively named choreas due to slow, non-patterned involuntary movements.
Staff & Alumni
Our team is made of investigators, researchers, Post-doctorate associates, and those passionate about medical research & advancement.