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    • 1. 发明申请
    • COMPOUNDS AND METHODS FOR THE TREATMENT OF LEUKEMIAS
    • 用于治疗白血病的化合物和方法
    • WO1994013793A1
    • 1994-06-23
    • PCT/US1993011144
    • 1993-11-16
    • APOLLON, INC.PACHUK, Catherine, J.CONEY, Leslie, R.OAKES, Fred, T.
    • APOLLON, INC.
    • C12N15/00
    • C12N15/113C07K2319/00C12N15/1135C12N2310/111C12N2310/121C12N2310/315C12N2310/351C12N2310/353C12N2310/53
    • Ribozymes are provided in which the ribozymes have a catalytic sequence, two legs and at least one anchor sequence complementary to the substrate mRNA at a location that can be noncontiguous with the portions of the substrate mRNA complementary to the legs. In certain preferred embodiments, ribozymes capable of cleaving the L6 mRNA or both the L6 and K28 mRNAs are provided. Methods are provided for the treatment of chronic myelogenous leukemia (CML) and acute lymphoblastic leukemia (ALL) patients having the L6 and K28 translocations and, therefore, the L6 mRNA. Additionally, combination treatments comprising components specific for both the K28 and L6 mRNAs are provided. Combination treatments are also provided for those patients which express c-myc in combination with K28 and/or L6 mRNAs. Methods of treatment include, for example, administration of ribozymes and antisense oligonucleotides directed against these mRNAs.
    • 提供了核酶,其中核酶具有催化序列,两条腿和至少一条与底物mRNA互补的锚定序列,所述锚定序列可以与与腿互补的底物mRNA的部分不连续的位置。 在某些优选实施方案中,提供能够切割L6 mRNA或L6和K28 mRNA两者的核酶。 提供了用于治疗慢性骨髓性白血病(CML)和具有L6和K28易位的急性淋巴细胞性白血病(ALL)患者以及L6mRNA的方法。 另外,提供包含对K28和L6 mRNA特异性成分的组合处理。 还为那些表达c-myc与K28和/或L6mRNA组合的患者提供组合治疗。 治疗方法包括例如施用针对这些mRNA的核酶和反义寡核苷酸。