Engineered meganucleases specific for recognition sequences in the hepatitis B virus genome
AssigneePrecision BioSciences, Inc.
InventorsDerek Jantz, James Jefferson Smith
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Abstract
The present invention encompasses engineered meganucleases which recognize and cleave a recognition sequence within an open reading frame (ORF) of the genome of at least two genotypes of the Hepatitis B virus (HBV). The present invention also encompasses methods of using such engineered meganucleases in a pharmaceutical composition and in methods for treating or reducing the symptoms of a HBV infection, or treating hepatocellular carcinoma (HCC). Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating HBV infections or HCC.
Claims13
A recombinant adeno-associated viral (AAV) vector comprising a polynucleotide, wherein said polynucleotide comprises a nucleic acid sequence encoding an engineered meganuclease,
- wherein said engineered meganuclease binds and cleaves a recognition sequence consisting of SEQ ID NO:
- 16,
- wherein said engineered meganuclease comprises a first subunit and a second subunit,
- wherein said first subunit binds a first recognition half-site of said recognition sequence,
- wherein said second subunit binds a second recognition half-site of said recognition sequence,
- wherein said engineered meganuclease comprises an amino acid sequence having at least 96% sequence identity to SEQ ID NO:
- 33,
- and wherein said nucleic acid sequence encoding said engineered meganuclease is operably linked to a promoter.
Dependencies are inferred from the claim text.