Active Research

Tay-Sachs & Sandhoff Diseases

Among all of the lysosomal storage disorders, the GM2 gangliosidoses are the most investigated subcategory. They are caused by the impaired degradation of GM2 ganglioside, a reaction which...

GM1-Gangliosidosis

GM1-gangliosidosis is an autosomal recession disease caused by mutations in the GLB1 gene encoding for the lysosomal acid beta-galactosidase (Bgal) enzyme. The resulting enzymatic deficiency...

AAV Vector Development

The Sena-Esteves laboratory aims to improve current limitations to AAV-based gene therapy for CNS diseases in two ways: 1) manipulation of the blood brain barrier (BBB) for increased...

Huntington Disease

HD is the most common among those movement diseases collectively named choreas. The term “chorea” (meaning dance) was borrowed from the ancient Greek theatre to describe the slow, non-patterned involuntary...