Aav5 capsid with non-canonical amino acid incorporation and uses thereof
AssigneeUniversity of Massachusetts
InventorsDan Wang, Hao Chang, Ailing Du, Guangping Gao
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AssigneeUniversity of Massachusetts
InventorsDan Wang, Hao Chang, Ailing Du, Guangping Gao
Estimating the term…
Aspects of the disclosure relate to compositions and methods for delivering a transgene (e.g., a transgene encoding one or more gene products) to a target cell. The disclosure is based, in part, on adeno-associated virus (AAV) capsid protein variants characterized by tropisms for certain cell types (e.g., neurons, lung cells muscle cells, bone cells, heart cells, etc.). In some embodiments, recombinant AAVs (rAAVs) comprising the capsid protein variants described herein exhibit enhanced lung tropism when compared to rAAVs having certain wild-type AAV capsid proteins (e.g., AAV5). Methods of delivering an rAAV comprising the AAV capsid protein variants are also described by the disclosure.
A modified adeno-associated virus (AAV) capsid protein comprising an amino acid sequence having one or more non-canonical amino acids (ncAA) incorporated into one or more variable regions (VRs) of the capsid protein.
A host cell comprising an rAAV vector expressing one or more of the following:
A rAAV comprising:
Dependencies are inferred from the claim text.