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发明公开
CN108124453A 用于将DNA序列靶向并入细胞或生物体的基因组中的Cas9逆转录病毒整合酶和Cas9重组酶系统 有权

用于将DNA序列靶向并入细胞或生物体的基因组中的Cas9逆转录病毒整合酶和Cas9重组酶系统
摘要：
本披露涉及使用与病毒整合酶、重组酶或转座酶附接的工程化蛋白如Cas9、Cpf1、TALE和锌指蛋白以便将感兴趣的DNA序列(或感兴趣的基因)递送至细胞或生物体的基因组中的靶向位点。使用在切割DNA的功能上无活性的Cas9将允许使用Cas9蛋白靶向DNA的能力，通过使用RNA引导物而不引起如在其他系统中预期的DNA断裂用于同源重组。还披露了使用与病毒整合酶或重组酶附接的锌指蛋白或TALE(结合特定DNA序列的工程化蛋白)。所述系统可用于实验室和治疗目的。例如，感兴趣的基因可以被包括在具有如下基因的细胞中，所述基因缺乏产生其基因产物以恢复所述细胞中的正常基因产物(例如基因产物可以是蛋白质或专用RNA)的能力。
摘要（英）：

The instant disclosure relates to the use of engineered proteins such as Cas9, Cpf1, TALE and Zinc finger proteins attached with a viral integrases, recombinase, or transposase in order to deliver a DNA sequence of interest (or gene of interest) to a targeted site in a genome of a cell or organism. The use of a Cas9 that is inactive for its function in cutting DNA will allow the use of Cas9 proteins ability to target DNA by the use of RNA guides without causing DNA breaks as intended in other systems for homologous recombination. The use of zinc finger proteins or TALE (engineered proteins that bind specific sequences of DNA) attached to the viral integrase or the recombinase is also disclosed. The system may be used for laboratory and therapeutic purposes. A gene of interest can be included in a cell with a gene lacking the ability to produce its gene product to recover the normal gene product in the cell (e.g. gene product may be a protein or specialized RNA).

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