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Enhancing the clinical coverage and anticancer efficacy of immune checkpoint blockade through manipulation of the gut microbiota

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Tay Sachs disease (GM2 Ganglioside) using targeted enzyme replacement therapy with alpha-hexosaminidase A or substrate inhibition of glucosylceramide synthase using imino sugars (e.g., miglustat (Zavesca marketed by Actelion) = N-butyl-deoxynojirimycin (NB-DNJ))

(1997) published findings in the Journal of Biological Chemistry demonstrating that IGF-1-mediated signaling through PI3K is essential for myoblast differentiation