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AssigneeShire Human Genetic Therapies, Inc.
InventorsVivian Choi, Xing Li
Estimating the term…
The present disclosure provides, among other things, a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid and a codon-optimized sequence encoding a human iduronate-2-sulfatase (I2S) enzyme. The disclosure also provides a method of treating a subject having Hunter syndrome (MPS II), comprising administering to the subject in need thereof a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid, and a promoter operably linked to a nucleic acid sequence that encodes iduronate-2-sulfatase (I2S), and wherein administering results in an increase in I2S enzymatic activity in the subject.
A recombinant adeno-associated virus (rAAV) vector comprising an AAV8 capsid or an AAV9 capsid, and a codon-optimized sequence encoding a human iduronate-2-sulfatase (I2S) enzyme.
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A recombinant adeno-associated virus (rAAV) comprising an AAV8 or AAV9 capsid and an rAAV vector, said vector comprising:
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A method of treating a subject having Hunter syndrome (MPS II), comprising administering to the subject in need thereof a rAAV of any one of the preceding claims.
A method of treating a subject having Hunter syndrome (MPS II), comprising administering to the subject in need thereof a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid, and a promoter operably linked to a nucleic acid sequence that encodes iduronate-2-sulfatase (I2S), and wherein administering results in an increase in I2S enzymatic activity in the subject.
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Dependencies are inferred from the claim text.