Raav-mediated nuclease-associated vector integration (raav-navi)
AssigneeUniversity of Massachusetts
InventorsAlexander Brown, Guangping Gao
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Abstract
Aspects of the disclosure relate to integration of a transgene packaged into recombinant adeno-associated virus (rAAV) by nuclease-assisted vector integration (NAVI).
Claims25
An isolated nucleic acid comprising at least one transgene flanked by inverted terminal repeats (ITRs), wherein the transgene is configured to be integrated into a target genome by nuclease-assisted vector integration, such that guide RNAs direct removal of the ITRs prior to transgene integration.
An isolated nucleic acid comprising an expression cassette engineered to express a first guide RNA (gRNA), wherein the expression cassette is flanked by inverted terminal repeats (ITRs), wherein the gRNA targets (e.g., hybridizes with) a nucleic acid sequence located adjacent to or within the nucleic acid sequence encoding the ITRs.
The rAAV of claim 9 or 10, wherein the at least one capsid protein is an AAV9 capsid protein.
Dependencies are inferred from the claim text.