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US 2020/0318081 A1Published application
Vectors with promoter and enhancer combinations for treating phenylketonuria
OwnerTyler Lahusen, Charles David Pauzainventors
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Abstract
A lentiviral vector system for expressing a lentiviral particle is disclosed. The lentiviral vector system includes a therapeutic vector. The lentiviral vector system produces a lentiviral particle for upregulating PAH expression in the cells of a subject afflicted with phenylketonuria (PKU).
Claims38
A viral vector comprising a therapeutic cargo portion, wherein the therapeutic cargo portion comprises:
- a PAH sequence or a variant thereof,
- a promoter;
- and
- a liver-specific enhancer,
- wherein the PAH sequence or the variant thereof is operatively controlled by both the promoter and the liver-specific enhancer.
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