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Well-tolerated and highly specific tailor-made recombinase for recombination of asymmetric target sites in multiple retrovirus strains

机译:耐受性强且高度特异性的量身定制重组酶,用于重组多种逆转录病毒株中的不对称靶位

摘要

The present invention relates to a method for preparing an expression vector encoding a well-tolerated and highly specific tailored recombinase, which tailored recombinase is capable of recombining asymmetric target sequences within the long terminal repeat (LTR) of proviral DNA of a plurality of retrovirus strains which may be inserted into the genome of a host cell, as well as to the obtained expression vector, cells transfected with these, expressed recombinase and pharmaceutical compositions comprising the expression vector, cells and/or recombinase. Pharmaceutical compositions are useful, e.g., in treatment and/or prevention of retrovirus infection, in particular, HIV infection. In particular, the invention relates to well-tolerated and highly specific tailored recombinases capable of combining asymmetric target sequences in a more than 90% of HIV-1 strains, thereby excising the HIV-1 sequences, and expression vectors encoding them.
机译:本发明涉及一种制备编码载体的方法,该表达载体编码耐受良好且高度特异性的重组酶,该重组酶能够在多个逆转录病毒株的前病毒DNA的长末端重复序列(LTR)内重组不对称靶序列。可将其插入宿主细胞的基因组以及所获得的表达载体中,将其转染的细胞表达重组酶以及包含该表达载体,细胞和/或重组酶的药物组合物。药物组合物可用于例如治疗和/或预防逆转录病毒感染,特别是HIV感染。特别地,本发明涉及能够在超过90%的HIV-1菌株中结合不对称靶序列,从而切除HIV-1序列的耐受良好且高度特异性的重组酶,以及编码它们的表达载体。

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