This enzyme is needed in healthy individuals for the process of hydrolysis of GM2 ganglioside to occur. For individuals with Tay-Sachs disease that lack this enzyme, the fatty substance of GM2 ...

In the human body, the highest concentration of sialic acid (as N-acetylneuraminic acid) occurs in the brain where it participates as an integral part of ganglioside structure in synaptogenesis ...

The integrity of this triad, which cooperate to hydrolyze ganglioside GM2 in the lysosome, is an absolute requirement; and when one or other of the genes is dysfunctional, GM2 ganglioside ...

The integrity of this triad, which cooperate to hydrolyse ganglioside GM2 in the lysosome, is an absolute requirement; and when one or other of the genes is dysfunctional, GM2 ganglioside ...

Vaccination with GM2/KLH-QS-21 stimulates the production of antibodies to the GM2 ganglioside, an antigen expressed by many melanomas. Serological response to GM2 was shown to be a positive ...

Furthermore, about 30% reductions in the concentration of secondary storage products GM2 and GM3 ganglioside, which are thought to be possible contributors to neuronal damage in lysosomal storage ...

In addition to the Rare Pediatric Disease designation, AXO-AAV-GM2 has Orphan Drug Designation (ODD) and is the first gene therapy that has been administered to children with Tay-Sachs disease.

Background: The gangliosides GM2, GD2, and GD3 are strongly expressed in sarcoma and represent novel antitumor targets. We have demonstrated the safety and immunogenicity of individual monovalent ...