Leading the worldwide fight to treat and cure
Tay-Sachs, Canavan, GM1 and Sandhoff diseases

2014 Research Initiative Grant

David Radin, PhD, Principal Investigator
BioStrategies, LC

Lectin-Assisted Transnasal Delivery of
Corrective Enzyme for GM1 Gangliosidosis


Radin and his coworkers tested the nasal administration of the in-house developed enzyme therapy in GM1 gangliosidosis mouse models. It was demonstrated that a small amount was detectable in the brain. However, the therapy was not delivered via olfactory nerves as designed, showed by assays on olfactory bulb of the mice. Further work was focused on understanding the mechanisms of trans-nasal delivery.


There are currently no effective treatments for GM1 gangliosidosis patients and Central Nervous System (CNS) delivery remains the fundamental barrier to Enzyme Replacement Therapy (ERT) strategies for these patients. There are different drug delivery routes available. Transnasal delivery, with its distinctive advantage of being convenient and non-invasive, offers potential of treating brain disorders like GM1. This research tested a lectin-based ERT carrier that may facilitate transnasal delivery of corrective β-gal to the brain in the GM1 mouse model. The results from this study will lay a foundation for further work on the intranasal enzyme therapy delivery for patients with GM1 and other neurodegenerative diseases.