Silencing of B4Galnt1 Gene Prevents GM2 Accumulation in Tay-Sachs Cells

Figure 1

Ganglioside biosynthesis and degradation pathway in Hexa-/-Neu3-/- TSD model. The dotted lines indicate the knock-out genes and proposed silencing of the B4Galnt1 gene by shRNA treatment (a). Schematic presentation of self-complementary recombinant AAV vector including shRNA silencing cassettes controlled by U6 promoter and mCherry reporter gene regulated by CMV promoter (b). Location of the target regions of the shRNA(s) for mouse and human B4Galnt1 gene (c).

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