SELECTIVE INSERTION OF SUICIDE GENES INTO CANCER CELLS

    公开(公告)号:WO2018217537A1

    公开(公告)日:2018-11-29

    申请号:PCT/US2018/033197

    申请日:2018-05-17

    IPC分类号: C12N15/63 C12N15/85

    摘要: A method for treating cancer by inducing a double strand DNA break at a chromosomal site in cancer cells, inserting a nucleic acid encoding a suicide gene into the chromosomal site having the double strand DNA break, and expressing the suicide gene in the cancer cells, thereby inducing cell death upon expression of the suicide gene. The chromosomal site includes a protospacer adjacent motif (PAM) absent from a corresponding chromosomal site in normal cells. Also disclosed is a cancer treatment method that includes identifying a single nucleotide change in the genomic sequence of cancer cells from a subject as compared to the genomic sequence of normal cells, the single nucleotide change forming a PAM, inserting a suicide gene specifically into the cancer cell genomic sequence, and activating the suicide gene, thereby leading to cell death. Furthermore, provided is a clustered regularly interspaced short palindromic repeat-associated protein having RNA-guided DNA endonuclease activity.

    吡咯喹啉醌衍生物及其组合物
    9.
    发明申请

    公开(公告)号:WO2018209601A1

    公开(公告)日:2018-11-22

    申请号:PCT/CN2017/084726

    申请日:2017-05-17

    发明人: 温传俊 孙奋勇

    摘要: 本发明涉及一种式I的吡咯喹啉醌衍生物或其药学上可接受的盐,其中,U、V、W、X、Y、Z各自独立取值为选自C、N、O、S的原子并连接在一起构成取代的芳香或非芳香环,条件是U不是O或S原子并且U、V、W、X、Y、Z满足各自取值的原子的化合价要求,不足部分与氢原子成键补足。所述吡咯喹啉醌衍生物或其药学上可接受的盐及其组合物,具有显著的抑癌作用,并且药物浓度较高的处理组抑癌效果更加明显,能将肿瘤细胞的生长活力抑制到30%-80%;同时,本发明式I的吡咯喹啉醌衍生物对自身免疫性疾病如红斑狼疮、代谢性疾病如Ⅱ型糖尿病和非酒精性脂肪性肝病、神经退行性疾病如阿尔兹海默病、炎症细胞过度活化所导致的疾病如感染性休克、细胞免疫疗法所导致的疾病如炎症风暴也有良好的治疗作用。