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    • 8. 发明授权
    • Use of ribozymes for functionating genes
    • 使用核酶作功能基因
    • US06803194B1
    • 2004-10-12
    • US09601970
    • 2000-08-09
    • James G. KeckJustin G. P. Wong
    • James G. KeckJustin G. P. Wong
    • C12Q168
    • C12N15/113C12N15/1034C12N2310/111C12N2310/121C12N2799/021C12N2799/027
    • Double stranded DNAs, expression vectors and methods for their use are provided in which the intracellular expression of the double stranded DNAs is used to alter the phenotype of a target cell so that the function of a target nucleic acid that includes a nucleotide sequence encoding a motif of interest can be determined using a combinatorial ribozyme library. The members of the library are catalytic RNAs that disrupt the expression of the transcription product of the target nucleic acid. Disruption of transcription product expression results in an altered cell phenotype which is used to determine the function of the target nucleic acid. The specific phenotype or response may be associated with normal cellular processes, or it may contribute to the generation of pathogenesis involved in disease development. The compositions find use in high-throughput screens to assign gene functions. When associated with a pathogenic phenotype, these genes or their gene products can constitute therapeutic targets for treatment of diseases. The complete sequence of the gene containing the target nucleic acid need not to be known for the method to be used successfully.
    • 提供双链DNA,表达载体及其使用方法,其中双链DNA的细胞内表达用于改变靶细胞的表型,使得靶核酸的功能包括编码基序的核苷酸序列 可以使用组合核酶库来确定感兴趣的。 文库的成员是破坏靶核酸转录产物表达的催化RNA。 转录产物表达的破坏导致改变的细胞表型,其用于确定靶核酸的功能。 特异性表型或反应可能与正常细胞过程相关,或可能有助于疾病发展中涉及的发病机制的产生。 该组合物可用于高通量筛选以分配基因功能。 当与致病表型相关时,这些基因或其基因产物可构成治疗疾病的治疗靶点。 含有目标核酸的基因的完整序列不必为成功使用的方法而知道。