Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome
AssigneePrecision BioSciences, Inc.
InventorsJames Jefferson Smith, Janel Lape, Victor Bartsevich, Hui Li
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Abstract
The present invention encompasses engineered nucleases which recognize and cleave a recognition sequence within a Hepatitis B virus (HBV) genome. The engineered meganucleases can exhibit at least one optimized characteristic, such as enhanced specificity and/or efficiency of indel formation, when compared to the first-generation meganuclease HBV 11-12×.26. 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 hepatocellular carcinoma.
Claims6
An engineered meganuclease that recognizes and cleaves a recognition sequence consisting of SEQ ID NO:
- 10 within a Hepatitis B virus genome, wherein said engineered meganuclease comprises a first subunit and a second subunit, wherein said first subunit binds to a first recognition half-site of said recognition sequence and comprises a first hypervariable (HVR1) region, wherein said second subunit binds to a second recognition half-site of said recognition sequence and comprises a second hypervariable (HVR2) region, wherein said engineered meganuclease comprises the amino acid sequence of SEQ ID NO:
- 12 or 13.
Dependencies are inferred from the claim text.