公开(公告)号：US20100196335A1

公开(公告)日：2010-08-05

申请号：US11919267

申请日：2006-04-28

申请人： Richard J. Samulski ,  Kyson Xiaohuai Chou

发明人： Richard J. Samulski ,  Kyson Xiaohuai Chou

IPC分类号： A61K35/12 ,  C07H21/02 ,  C12N15/74 ,  C12N5/10 ,  A61K31/7088 ,  C12P21/00 ,  C12P19/34 ,  A61K31/7105 ,  C12Q1/68 ,  A61P43/00

CPC分类号： C12N15/85 ,  C12N15/111 ,  C12N15/67 ,  C12N15/8509 ,  C12N2310/11 ,  C12N2320/33 ,  C12N2840/44 ,  C12N2840/445

摘要： The present invention provides an isolated nucleic acid comprising: a) at least one first nucleotide sequence encoding a heterologous nucleotide sequence of interest; and b) at least two second heterologous nucleotide sequences, wherein each second heterologous nucleotide sequences comprises: i) a first set of splice elements defining a first intron that is removed by splicing to produce a first RNA molecule that imparts a biological function in the absence of activity at a second set of splice elements; and ii) the second set of splice elements defining one or more introns different from said first intron, wherein said one or more introns different from said first intron are removed by splicing to produce no RNA molecule and/or a second RNA molecule that docs not impart a biological function, when said second set of splice elements is active. Further provided are methods of using the nucleic acid of this invention to regulate transgene expression.

摘要翻译： 本发明提供了一种分离的核酸，其包含：a）至少一个编码感兴趣的异源核苷酸序列的第一核苷酸序列; 和b）至少两个第二异源核苷酸序列，其中每个第二异源核苷酸序列包括：i）限定第一内含子的第一组剪接元件，其通过剪接除去以产生在不存在时赋予生物学功能的第一RNA分子 在第二组拼接元件上的活动; 和ii）限定与所述第一内含子不同的一个或多个内含子的第二组拼接元件，其中与所述第一内含子不同的所述一个或多个内含子通过剪接除去以产生不分泌RNA的RNA分子和/或第二RNA分子 当所述第二组接头元件是活动的时，赋予生物功能。 还提供了使用本发明的核酸调节转基因表达的方法。

公开(公告)号：US20100303776A1

公开(公告)日：2010-12-02

申请号：US12799095

申请日：2010-04-16

申请人： Richard Jude Samulski ,  Kyson Xiaohuai Chou

发明人： Richard Jude Samulski ,  Kyson Xiaohuai Chou

IPC分类号： A61K35/12 ,  C12N5/10 ,  C07H21/02 ,  C12N15/63 ,  A61K31/7105 ,  C12P19/34 ,  A61P43/00

CPC分类号： C12N15/63 ,  C12N15/111 ,  C12N15/85 ,  C12N2310/11 ,  C12N2310/12 ,  C12N2310/14 ,  C12N2330/51 ,  C12N2799/025 ,  C12N2830/42 ,  C12N2840/44

摘要： The present invention provides a cell comprising a first heterologous nucleic acid construct and a second heterologous nucleic acid construct, wherein each of said first and second heterologous nucleic acid constructs comprises: A. a first nucleotide sequence encoding a nucleotide sequence of interest (NOI); and B. a second intronic nucleotide sequence comprising: i) a first set of splice elements defining a first intron that is removed by splicing to produce a first RNA molecule encoded by said first nucleotide sequence in the absence of activity at a second set of splice elements; and ii) the second set of splice elements defining a second intron different from said first intron, wherein said second intron is removed by splicing to produce no RNA molecule and/or to produce a second RNA molecule that is not encoded by said first nucleotide sequence, wherein each said first nucleotide sequence of each of said first and second heterologous nucleic acid constructs is different from one another and wherein each said second intronic nucleotide sequence of each of said first and second heterologous nucleic acid constructs is different from one another.

摘要翻译： 本发明提供了包含第一异源核酸构建体和第二异源核酸构建体的细胞，其中所述第一和第二异源核酸构建体中的每一个包含：A.编码目的核苷酸序列（NOI）的第一核苷酸序列; B.一种第二内含子核苷酸序列，其包含：i）限定第一内含子的第一组剪接元件，其通过剪接除去以在第二组剪接下不存在活性产生由所述第一核苷酸序列编码的第一RNA分子 元素 和ii）限定与所述第一内含子不同的第二内含子的第二组剪接元件，其中通过剪接除去所述第二内含子以产生RNA分子和/或产生不由所述第一核苷酸序列编码的第二RNA分子 其中每个所述第一和第二异源核酸构建体的每个所述第一核苷酸序列彼此不同，并且其中每个所述第一和第二异源核酸构建体的每个所述第二内含子核苷酸序列彼此不同。