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    • 1. 发明申请
    • ALLELE EDITING AND APPLICATIONS THEREOF
    • ALLELE编辑和应用程序
    • WO2017186718A1
    • 2017-11-02
    • PCT/EP2017/059799
    • 2017-04-25
    • UNIVERSITÄT BASEL
    • KORNETE, MaraJEKER, Lukas
    • C12N15/10C12N15/90C12Q1/68
    • The invention relates to a method to determine a homology directed repair (HDR) event within a eukaryotic cell, wherein the cell expresses a first isoform of a surface protein, which is different from a second isoform of said surface protein with regard to an amino acid marker. The method comprises the steps of inducing a DNA double strand break, providing a HDR template DNA construct comprising the amino acid marker corresponding to the second isoform of the surface protein and subsequently determining the expression of the first or second isoform of said surface protein on said cell, wherein expression of the second isoform indicates a successful HDR event. The invention also relates to a method for editing a genomic location of interest within a eukaryotic cell, and to a method of selectively depleting or enriching an edited cell in a composition of non-edited and edited cells.
    • 本发明涉及确定真核细胞内的同源性定向修复(HDR)事件的方法,其中所述细胞表达表面蛋白的第一同种型,所述第一同种型不同于所述表达蛋白的第二同种型 表面蛋白关于氨基酸标记。 该方法包括以下步骤:诱导DNA双链断裂,提供包含对应于表面蛋白的第二同种型的氨基酸标记的HDR模板DNA构建体,随后确定所述表面蛋白的第一或第二同种型在所述 细胞,其中第二同种型的表达指示成功的HDR事件。 本发明还涉及用于编辑真核细胞内的感兴趣的基因组位置的方法,并且涉及选择性地消耗或富集未编辑和编辑的细胞的组合物中的编辑的细胞的方法。
    • 2. 发明申请
    • IMMUNOLOGICALLY DISCERNIBLE CELL SURFACE VARIANTS FOR USE IN CELL THERAPY
    • 免疫学上可用的细胞表面变体用于细胞治疗
    • WO2018083071A1
    • 2018-05-11
    • PCT/EP2017/077826
    • 2017-10-30
    • UNIVERSITÄT BASEL
    • JEKER, LukasKORNETE, MaraBORDOLI SCHWEDE, LorenzaSCHWEDE, TorstenLEPORE, RosalbaMATTER MARONE, RominaRECHER, Mike
    • C12N15/10C12N15/90C12Q1/68A61K35/12C07K14/705
    • The invention relates to a mammalian cell, particularly a human cell, expressing a first isoform of a surface protein, wherein the first isoform is functionally indistinguishable, but immunologically distinguishable from a second isoform, for use in a medical treatment of a patient having cells expressing the second isoform form of the surface protein. The invention further relates to an agent selected from 1) a compound comprising, or consisting of, an antibody or antibody-like molecule and 2) an immune effector cell bearing an antibody-like molecule or an immune effector cell bearing a chimeric antigen receptor, for use in a method of treatment of a medical condition, wherein the agent is specifically reactive to either a first or a second isoform of a surface protein, wherein the first isoform is functionally indistinguishable, but immunologically distinguishable from the second isoform, and wherein the agent is administered to ablate a cell bearing the isoform that the agent is reactive to.
    • 本发明涉及表达表面蛋白的第一同种型的哺乳动物细胞,特别是人细胞,其中第一同种型在功能上不可区分,但在免疫学上与第二同种型可区分,用于 对表达蛋白质的第二同种型形式的细胞进行治疗。 本发明还涉及选自以下的药剂:1)包含抗体或抗体样分子或由其组成的化合物和2)携带抗体样分子的免疫效应细胞或带有嵌合抗原受体的免疫效应细胞, 用于治疗医学病症的方法中,其中所述药剂对表面蛋白的第一或第二同种型具有特异性反应性,其中所述第一同种型在功能上不可区分,但免疫学上可区别于第二同种型,并且其中所述 施用药剂以消融具有药剂对其反应的同种型的细胞。