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    ANTISENSE COMPOUNDS TARGETING GENES ASSOCIATED WITH USHER SYNDROME
    2.
    发明申请
    ANTISENSE COMPOUNDS TARGETING GENES ASSOCIATED WITH USHER SYNDROME 有权
    抗体化合物靶向基因与USHER综合征相关

    公开(公告)号：US20140114057A1

    公开(公告)日：2014-04-24

    申请号：US14009782

    申请日：2012-05-02

    申请人： Michelle L. Hastings ,  Frank Rigo ,  C. Frank Bennett

    发明人： Michelle L. Hastings ,  Frank Rigo ,  C. Frank Bennett

    IPC分类号： C12N15/113

    CPC分类号： C12N15/113 ,  C07H21/04 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/33 ,  C12N2310/3525

    摘要： The present invention provides compounds comprising oligonucleotides complementary to an Usher transcript. Certain such compounds are useful for hybridizing to an Usher transcript, including but not limited, to an Usher transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the Usher transcript. In certain such embodiments, the Usher transcript includes a mutation that results in cryptic splicing and hybridization of the oligonucleotide results in a decrease in the amount of cryptic splicing. In certain embodiments, such compounds are used to treat Usher Syndrome.

    摘要翻译： 本发明提供了包含与Usher转录物互补的寡核苷酸的化合物。 某些这样的化合物可用于与Usher转录物杂交，包括但不限于细胞中的Usher转录物。 在某些实施方案中，这种杂交导致Usher转录物的剪接的调节。 在某些这样的实施方案中，Usher转录物包括导致隐含剪接的突变，并且寡核苷酸的杂交导致隐形剪接的量的减少。 在某些实施方案中，这些化合物用于治疗Usher综合征。

    Antisense compounds targeting genes associated with usher syndrome
    4.
    发明授权
    Antisense compounds targeting genes associated with usher syndrome 有权
    靶向与引发综合征相关的基因的反义化合物

    公开(公告)号：US09353371B2

    公开(公告)日：2016-05-31

    申请号：US14009782

    申请日：2012-05-02

    申请人： Michelle L. Hastings ,  Frank Rigo ,  C. Frank Bennett

    发明人： Michelle L. Hastings ,  Frank Rigo ,  C. Frank Bennett

    IPC分类号： C12N15/113

    CPC分类号： C12N15/113 ,  C07H21/04 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/33 ,  C12N2310/3525

    摘要： The present invention provides compounds comprising oligonucleotides complementary to an Usher transcript. Certain such compounds are useful for hybridizing to an Usher transcript, including but not limited, to an Usher transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the Usher transcript. In certain such embodiments, the Usher transcript includes a mutation that results in cryptic splicing and hybridization of the oligonucleotide results in a decrease in the amount of cryptic splicing. In certain embodiments, such compounds are used to treat Usher Syndrome.

    摘要翻译： 本发明提供了包含与Usher转录物互补的寡核苷酸的化合物。 某些这样的化合物可用于与Usher转录物杂交，包括但不限于细胞中的Usher转录物。 在某些实施方案中，这种杂交导致Usher转录物的剪接的调节。 在某些这样的实施方案中，Usher转录物包括导致隐含剪接的突变，并且寡核苷酸的杂交导致隐形剪接的量的减少。 在某些实施方案中，这些化合物用于治疗Usher综合征。