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    • 92. 发明公开
    • TAL EFFECTOR MEANS USEFUL FOR PARTIAL OR FULL DELETION OF DNA TANDEM REPEATS
    • TAL-EFFEKTORMITTEL ZUR PARTIELLEN ODERVOLLSTÄNDIGEN删除VON DNA-TANDEM-REPEATS
    • EP3074512A1
    • 2016-10-05
    • EP14838781.4
    • 2014-11-26
    • Institut Pasteur
    • RICHARD, Guy-FranckMOSBACH, ValentineVITERBO, David
    • C12N9/22C12N15/10C12N15/62A61P25/00A61P21/00A61P19/00A61K38/16A61K48/00
    • C12N9/22A61K48/005C07K2319/80C12N15/62C12N15/907C12Y301/21004
    • The application relates to means, which derive from TAL effectors and TALENs. The structure of the means of the application is especially adapted for partial or full deletion of at least one DNA tandem repeat, more particularly for partial or full deletion of at least one DNA tandem repeat in a double-stranded DNA, more particularly for partial or full deletion of at least one DNA tandem repeat, which is contained in a double-stranded DNA and, which forms a complex secondary structure, such as a hairpin, a triple helix or a tetraplex secondary structure. The means of the application are notably useful in the treatment and/or prevention and/or palliation of a disease or disorder involving at least one DNA tandem repeat, such as DM1, SCA8, SCA12, HDL2, SBMA, HD, DRPLA, SCA1, SCA2, SCA3, SCA6, SCA7, SCA17, PSACH, DM2, SCA10, SPD1, OPMD, CCD, HPE5, HFG syndrome, BPES, EIRE1, FRAXA, FXTAS and FRAXE.
    • 本申请涉及从TAL效应器和TALEN得到的方法。 所述应用手段的结构特别适用于部分或完全缺失至少一个DNA串联重复,更特别地适用于双链DNA中至少一个DNA串联重复的部分或完全缺失,更特别是部分或全部 至少一个DNA串联重复的完全缺失,其包含在双链DNA中,并形成复合二级结构,例如发夹,三螺旋或四倍体二级结构。 本申请的方法在治疗和/或预防和/或缓解涉及至少一种DNA串联重复的疾病或病症中非常有用,例如DM1,SCA8,SCA12,HDL2,SBMA,HD,DRPLA,SCA1, SCA2,SCA3,SCA6,SCA7,SCA17,PSACH,DM2,SCA10,SPD1,OPMD,CCD,HPE5,HFG综合征,BPES,EIRE1,FRAXA,FXTAS和FRAXE。