THERAPEUTIC mRNA

Abstract: the present invention relates to mrna, mrna compositions, kits and uses thereof for the treatment of lysosomal storage diseases. in one aspect, the present invention relates to the treatment of niemann-pick type c disease.

Inventor(s): Denzil FURTADO, Frank CARUSO

CPC Classification: A61K48/005 (Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy)

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