Do you think this will one day be attempted in humans If so when

rumanaqw

[i=s] 本帖最后由 rumanaqw 于 2023-9-16 18:08 编辑 [/i]

Waddington: This was gene supplementation – delivering extra working copies of the  glucocerebrosidase (GBA) gene, which encodes the lysosomal enzyme that is deficient in Gaucher's disease. We delivered the genes using a gene therapy vector, a delivery vehicle developed based on viral biology (viruses are great at delivering genetic material).

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RG: What's significant about this study? Waddington: The unique aspects of our study were the fact that we delivered the therapeutic gene in utero to prevent the neurodegenerative disease from ever occurring. We demonstrated that this vector could be delivered, and result in widespread gene expression to the larger, non-human primate brain.

There are two major take-home points: First, we provide evidence that Gaucher Disease may be treatable, even curable, by gene therapy and that even the acute neuronopathic disease at the extreme end of the Gaucher Disease spectrum may be treatable. Second, we have performed several experiments to demonstrate the feasibility of fetal gene therapy that could be translated to humans.