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    • 2. 发明申请
    • ALPHAVIRUS cDNA VECTORS
    • WO1995027044A1
    • 1995-10-12
    • PCT/SE1995000343
    • 1995-03-30
    • BIOPTION ABLILJESTRÖM, PeterGAROFF, Henrik
    • BIOPTION AB
    • C12N07/01
    • C12N15/113A61K38/00A61K48/00C12N15/86C12N2310/111C12N2310/123C12N2770/36143C12N2830/52
    • The present invention is related to polynucleotide molecules and to their use for production of desired products after introduction into human or animal cells. In addition, the present invention is concerned with pharmaceutical compositions comprising said polynucleotide molecules and their use in prophylactic or therapeutic treatment methods. The present invention is also related to use of such polynucleotide molecules in animals to achieve expression of desired products, which can be recovered from the animal but do not give rise to any beneficial, e.g. therapeutical, activity in the said animal. More specifically, the present invention is directed to alphavirus cDNA vectors comprised of recombinant cDNA consisting of cDNA derived from an alphavirus and heterologous, i.e. foreign, cDNA coding for a desired substance.
    • 本发明涉及多核苷酸分子及其在引入人或动物细胞后产生所需产物的用途。 此外,本发明涉及包含所述多核苷酸分子的药物组合物及其在预防或治疗方法中的用途。 本发明还涉及在动物中使用这种多核苷酸分子以实现所需产物的表达,其可以从动物中回收,但不产生任何有益的,例如, 所述动物的治疗活性。 更具体地,本发明涉及包含由源自甲病毒属的cDNA和异源(即外来的)编码所需物质的cDNA的cDNA组成的重组cDNA的甲病毒cDNA载体。
    • 4. 发明申请
    • RIBOZYMES TARGETING THE RETROVIRAL PACKAGING SEQUENCE EXPRESSION CONSTRUCTS AND RECOMBINANT RETROVIRUSES CONTAINING SUCH CONSTRUCTS
    • 重组包装序列表达构建体和包含这些构建体的重组逆转录酶的RIBOZYMES
    • WO1996022368A1
    • 1996-07-25
    • PCT/AU1996000022
    • 1996-01-18
    • GENE SHEARS PTY. LTD.SYMONDS, Geoffrey, P.SUN, Lun-Quan
    • GENE SHEARS PTY. LTD.
    • C12N15/11
    • C12N15/1132A61K38/00C12N15/1131C12N2310/111C12N2310/12C12N2310/121C12N2310/122C12N2310/123C12N2310/126C12N2310/13C12N2799/027
    • This invention is directed to a synthetic non-naturally occurring oligonucleotide compound which comprises nucleotides whose sequence defines a conserved catalytic region and nucleotides whose sequence is capable of hybridizing with a predetermined target sequence within a packaging sequence of an RNA virus. Preferably, the viral packaging sequence is a retrovirus packaging sequence or the HIV-1 Psi packaging sequence. The RNA virus may be HIV-1, Feline Leukemia Virus, Feline Immunodeficiency Virus or one of the viruses listed in Table (I). The conserved catalytic region may be derived from a hammerhead ribozyme, a hairpin ribozyme, a hepatitis delta ribozyme, an RNAase P ribozyme, a group (I) intron, a group (II) intron. The invention is also directed to multiple ribozymes, combinations of ribozymes, with or without antisense, and combinations of ribozymes, with antisense, and TAR decoys, polyTARs and RRE decoys targeted against the RNA virus. Vectors are also described. Further, methods of treatment and methods of use both in vivo and ex vivo are described.
    • 本发明涉及合成的非天然存在的寡核苷酸化合物,其包含其序列限定保守催化区的核苷酸和其序列能够与RNA病毒的包装序列内的预定靶序列杂交的核苷酸。 优选地,病毒包装序列是逆转录病毒包装序列或HIV-1 Psi包装序列。 RNA病毒可以是HIV-1,猫白血病病毒,猫免疫缺陷病毒或表(I)中列出的病毒之一。 保守的催化区域可以衍生自锤头核酶,发夹核酶,戊型肝炎核酶,RNA酶P核酶,组(I)内含子,组(II)内含子。 本发明还涉及多种核酶,具有或不具有反义的核酶的组合,以及核酶与反义和TAR诱饵,针对RNA病毒靶向的多核苷酸和RRE诱饵的组合。 还描述了载体。 此外,描述了体内和体外的治疗方法和使用方法。