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US 12,577,539 B2Granted
Cells for enhanced production of adeno-associated virus
AssigneeThe Regents of the University of California
InventorsDavid V. Schaffer, Christopher Barnes, David Stephen Ojala
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Abstract
The present disclosure provides an in vitro mammalian cell that is genetically modified to provide for enhanced production of adeno-associated virus (AAV) virions. The mammalian cells can be used to produce AAV virions, e.g., recombinant AAV virions that include a heterologous nucleic acid encoding a gene product; the present disclosure thus provides methods for producing an AAV virion, which may be a recombinant AAV virion.
Claims10
An in vitro mammalian cell comprising:
- (i) one or more genetic modifications that provide for increased adeno-associated virus (AAV) virion production by the cell, wherein the genetic modification comprises:
- integration into the genome of the cell of a nucleic acid comprising a nucleotide sequence encoding the Spindle and Kinetochore Associated Complex subunit 2 (SKA2) polypeptide and/or the Inositol Tri-Phosphate Receptor Interacting Protein (ITPRIP2) polypeptide and results in increased production of the SKA2 polypeptide and/or the ITPRIP2 polypeptide, respectively, compared to a control mammalian cell not comprising the one or more genetic modifications;
- and
- (ii) a recombinant adeno-associated virus (AAV) genome comprising a heterologous nucleic acid encoding one or more heterologous gene products,
- wherein the cell, when provided with helper functions, packages the AAV genome into an AAV virion, and produces the AAV virion at a level that is higher than the level of AAV virion produced by a control cell.
2 more elements
Dependencies are inferred from the claim text.