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Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome
Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome
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机译:优化的工程官核酸核糖核糖酶在乙型肝炎病毒基因组中具有特异性识别序列
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摘要
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.
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