One-Year Progress Report to National Tay-Sachs & Allied Diseases Association
Lectin-assisted transnasal delivery of corrective enzyme for GM1 gangliosidosis
Investigators: David N. Radin, Carole L Cramer, Alessandra d'Azzo (SJCRH)
Genetic deficiencies in the lysosomal enzyme β-galactosidase (β-gal) lead to the neurodegenerative disease GM1 gangliosidosis. Traditional enzyme replacement therapy (ERT) approaches are ineffective because they fail to deliver sufficient enzyme to the brain. Our goal in this research, is to determine if the plant RTB lectin (sugar-binding protein) can help to carry β-gal across nasal mucosal surfaces and increase access to the brain using the mouse model of GM1. Fusion proteins linking β-gal with the RTB lectin will be produced in a plant-based protein production system and the purified protein will be administered trans-nasally to β-gal-/- mice to determine if active enzyme reaches the cells of the brain.