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Genetically engineered meganuclease specific for the recognition sequence of the hepatitis B virus genome

机译:基因工程的Meganuclease特异于甲型肝炎病毒基因组的识别序列

摘要

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.
机译:本发明包括识别和切割甲型乙型肝炎病毒(HBV)的至少两种基因型的基因组的开放阅读框(ORF)内识别和切割识别序列的工程化的Meganuclase。 本发明还包括在药物组合物中使用这种工程化美容核糖核糖酶的方法以及用于治疗或减少HBV感染的症状,或治疗肝细胞癌(HCC)的方法。 此外,本发明包括药物组合物,所述药物组合物包含工程化的Meganuclease蛋白,编码工程化的Meganucl酶的核酸,以及使用这种组合物来治疗HBV感染或HCC。

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