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LAGLIDADG homing endonuclease that cleaves CC chemokine receptor type 5 (CCR5) gene and its use

机译:切割CC趋化因子受体5型(CCR5)基因的LAGLIDADG归巢内切核酸酶及其用途

摘要

Disclosed herein are compositions for inactivating the human CCR5 gene comprising engineered LAGLIDADG homing endonucleases (LHEs) and their derivatives, particularly derived from members of the -Onul subfamily of LHE. Polynucleotides encoding such endonucleases, vectors comprising said polynucleotides, cells comprising or having been treated with such endonucleases, and therapeutic compositions deriving therefrom are also provided.
机译:本文公开了用于使人CCR5基因失活的组合物,其包含工程化的LAGLIDADG归巢内切核酸酶(LHE)及其衍生物,特别是衍生自LHE的Onul亚家族的成员。还提供了编码此类核酸内切酶的多核苷酸,包含所述多核苷酸的载体,包含此类内切核酸酶或已经用此类内切核酸酶处理的细胞以及由此衍生的治疗组合物。

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