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AssigneeTakeda Pharmaceutical Company Limited
InventorsMatthias KLUGMANN, Hanspeter ROTTENSTEINER, Franziska HORLING, Johannes LANGLER
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The present disclosure provides, among other things, a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 capsid and a codon-optimized sequence encoding a human phenylalanine hydroxylase (PAH) enzyme. The disclosure also provides a method of treating a subject having phenylketonuria (PKU), comprising administering to the subject in need thereof a recombinant adeno-associated vims (rAAV) vector comprising an AAV8 capsid, and a promoter operably linked to a nucleic acid sequence that encodes PAH, and wherein administering results in a decrease in phenylalanine level in the subject.
A recombinant adeno-associated virus vector (rAAV) comprising a codon-optimized sequence encoding a human phenylalanine hydroxylase (PAH), wherein the codon-optimized sequence has at least 70% identity to one of SEQ ID Nos:
The rAAV of any one of the preceding claims, wherein the rAAV encodes an AAV8 capsid.
The rAAV of any one of the preceding claims, wherein the rAAV further comprises a WPRE sequence.
The rAAV of claim of any one of the preceding claims, wherein the rAAV further comprises a liver-specific promoter.
The rAAV of any one of the preceding claims, wherein the rAAV comprises a cis-acting regulatory module (CRM).
The rAAV of any one of the preceding claims, wherein the rAAV further comprises an intron upstream of the PAH sequence.
A method of treating phenylketonuria (PKU), comprising administering to a subject in need of treatment a recombinant adeno-associated virus (rAAV) comprising a codon-optimized sequence encoding a human phenylalanine hydroxylase (PAH), wherein the codon-optimized sequence has at least 70% identity to one of SEQ ID Nos:
Dependencies are inferred from the claim text.