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    NUCLEIC ACID SILENCING SEQUENCES
    3.
    发明申请
    NUCLEIC ACID SILENCING SEQUENCES 有权
    核酸沉淀序列

    公开(公告)号：US20160264994A1

    公开(公告)日：2016-09-15

    申请号：US15074785

    申请日：2016-03-18

    申请人： University of Massachusetts

    发明人： Jeanne B. Lawrence ,  Lisa L. Hall

    IPC分类号： C12N15/85 ,  C12N15/113 ,  A61K48/00 ,  C12N15/90

    CPC分类号： C12N15/85 ,  A61K48/00 ,  A61K48/005 ,  C07K2319/81 ,  C12N9/22 ,  C12N15/113 ,  C12N15/86 ,  C12N15/907 ,  C12N2310/11 ,  C12N2320/32 ,  C12N2330/10 ,  C12N2750/14143

    摘要： The present invention features compositions and methods for introducing, into cells, nucleic acids whose expression results in chromosomal silencing. The nucleic acids are targeted to specific chromosomal regions where they subsequently reduce the expression of deleterious genes, or cause the death of deleterious cells. Where the nucleic acid sequence is a silencing sequence, it may encode an Xist RNA or other non-coding, silencing RNA. Accordingly, the present invention features, inter alia, nucleic acid constructs that include a transgene (e.g., a silencing sequence encoding an Xist RNA or other non-coding RNA that silences a segment of a chromosome); first and second sequences that direct insertion of the silencing sequence into a targeted chromosome; and, optionally, a selectable marker.

    摘要翻译： 本发明的特征在于将表达导致染色体沉默的核酸引入细胞的组合物和方法。 核酸靶向特定的染色体区域，其中它们随后减少有害基因的表达，或导致有害细胞的死亡。 当核酸序列是沉默序列时，其可以编码Xist RNA或其他非编码沉默RNA。 因此，本发明特别地包括包含转基因（例如，编码Xist RNA或沉默染色体片段的其他非编码RNA的沉默序列）的核酸构建体; 将沉默序列插入目标染色体的第一和第二序列; 和可选地，选择标记。

    NUCLEIC ACID SILENCING SEQUENCES
    5.
    发明申请
    NUCLEIC ACID SILENCING SEQUENCES 有权
    核酸沉淀序列

    公开(公告)号：US20140127810A1

    公开(公告)日：2014-05-08

    申请号：US14045057

    申请日：2013-10-03

    申请人： University of Massachusetts

    发明人： Jeanne B. Lawrence ,  Lisa L. Hall

    IPC分类号： C12N15/85

    CPC分类号： C12N15/85 ,  A61K48/00 ,  A61K48/005 ,  C07K2319/81 ,  C12N9/22 ,  C12N15/113 ,  C12N15/86 ,  C12N15/907 ,  C12N2310/11 ,  C12N2320/32 ,  C12N2330/10 ,  C12N2750/14143

    摘要： The present invention features compositions and methods for introducing, into cells, nucleic acids whose expression results in chromosomal silencing. The nucleic acids are targeted to specific chromosomal regions where they subsequently reduce the expression of deleterious genes, or cause the death of deleterious cells. Where the nucleic acid sequence is a silencing sequence, it may encode an Xist RNA or other non-coding, silencing RNA. Accordingly, the present invention features, inter alia, nucleic acid constructs that include a transgene (e.g., a silencing sequence encoding an Xist RNA or other non-coding RNA that silences a segment of a chromosome); first and second sequences that direct insertion of the silencing sequence into a targeted chromosome; and, optionally, a selectable marker.

    摘要翻译： 本发明的特征在于将表达导致染色体沉默的核酸引入细胞的组合物和方法。 核酸靶向特定的染色体区域，其中它们随后减少有害基因的表达，或导致有害细胞的死亡。 当核酸序列是沉默序列时，其可以编码Xist RNA或其他非编码沉默RNA。 因此，本发明特别地包括包含转基因（例如，编码Xist RNA或沉默染色体片段的其他非编码RNA的沉默序列）的核酸构建体; 将沉默序列插入目标染色体的第一和第二序列; 和可选地，选择标记。

    DOSAGE COMPENSATING TRANSGENES AND CELLS
    8.
    发明申请
    DOSAGE COMPENSATING TRANSGENES AND CELLS 有权
    剂量补充转基因和细胞

    公开(公告)号：US20140294785A1

    公开(公告)日：2014-10-02

    申请号：US14215997

    申请日：2014-03-17

    申请人： UNIVERSITY OF MASSACHUSETTS

    发明人： Jeanne B. Lawrence ,  Jun Jiang ,  Lisa L. Hall

    IPC分类号： C12N15/113

    CPC分类号： A01K67/0275 ,  C07K14/4703 ,  C12N5/0647 ,  C12N9/12 ,  C12N2510/00

    摘要： Methods and compositions for reducing expression of genes on Chromosome 21 (“Chr 21”) by targeting an XIST transgene to the Dual specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A) gene or a Regulator of calcineurin 1 (RCAN1) gene, and cells and transgenic animals comprising an XIST transgene inserted into a DYRK1A or RCAN1 allele, e.g., cells and animals trisomic for human Chr 21 and mouse Chr 16.

    摘要翻译： 通过将XIST转基因靶向双重特异性酪氨酸磷酸化调节激酶1A（DYRK1A）基因或钙调神经磷酸酶1（RCAN1）基因的调节剂）和细胞，减少染色体21（“Chr 21”）上基因表达的方法和组合物 以及包含插入到DYRK1A或RCAN1等位基因中的XIST转基因的转基因动物，例如用于人Chr 21和小鼠Chr 16的三体细胞和动物。