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AssigneesCANbridge Pharmaceuticals, Inc., LogicBio Therapeutics, Inc.
InventorsYunxiang Zhu, Peter Pechan, Anannya Banga
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The present disclosure provides compositions comprising isolated, e.g., recombinant, adeno-associated viruses (AAV) particles, comprising a liver tropic capsid protein, e.g., an sL65 capsid protein, for delivery of a GAA protein. The present disclosure also provides compositions comprising a first nucleic acid enoding a liver tropic capsid protein, e.g., an sL65 capsid protein, and a second nucleic acid comprising a transgene encoding a GAA protein. The present disclosure also provides methods for making an isolated, e.g., recombinant, AAV particles, and methods for deliverying an exogenous GAA protein into a subject and/or methods for treating a subject having a GAA-associated disease or disorder, e.g., a lysosomal storage disorder, e.g., Pompe disease.
An isolated recombinant adeno-associated virus (rAAV) particle comprising an AAV capsid protein, wherein the capsid protein comprises an amino acid sequence of SEQ ID NO:
A composition comprising a first nucleic acid encoding an AAV capsid protein, wherein the capsid protein comprises an amino acid sequence of SEQ ID NO:
An isolated nucleic acid comprising a transgene encoding an alpha-glucosidase (GAA) protein, wherein the transgene encoding the GAA protein comprises the nucleotide sequence of any one of SEQ ID NOs:
A method of making an isolated recombinant adeno-associated virus (rAAV) particle, the method comprising
A method of making an isolated recombinant adeno-associated virus (rAAV) particle, the method comprising
The method of embodiment 120 or 121, wherein the GAA-associated disease is a lysosomal storage disease.
An isolated recombinant adeno-associated virus (rAAV) particle comprising an AAV viral genome of any one of SEQ ID NO:
An isolated recombinant viral genome comprising or consisting of the nucleic acid sequence of any one of SEQ ID NO:
Dependencies are inferred from the claim text.