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5 条记录 (耗时 0.018 秒)

    Variant RNAi
    1.
    发明授权
    Variant RNAi 有权

    公开(公告)号：US10760079B2

    公开(公告)日：2020-09-01

    申请号：US16566565

    申请日：2019-09-10

    申请人： Genzyme Corporation

    发明人： Lisa M. Stanek ,  Adam Palermo ,  Brenda Richards ,  Sergio Pablo Sardi ,  Catherine O'Riordan ,  Antonius Song

    IPC分类号： C07H21/04 ,  C12N15/113

    摘要： Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.

    Variant RNAi
    2.
    发明授权
    Variant RNAi 有权

    公开(公告)号：US10450563B2

    公开(公告)日：2019-10-22

    申请号：US15549895

    申请日：2016-02-09

    申请人： Genzyme Corporation

    发明人： Lisa M. Stanek ,  Adam Palermo ,  Brenda Richards ,  Sergio Pablo Sardi ,  Catherine O'Riordan ,  Antonius Song

    IPC分类号： C07H21/04 ,  C12N15/113

    摘要： Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.

    Variant RNAi
    4.
    发明授权
    Variant RNAi 有权

    公开(公告)号：US11781137B2

    公开(公告)日：2023-10-10

    申请号：US16941303

    申请日：2020-07-28

    申请人： Genzyme Corporation

    发明人： Lisa M. Stanek ,  Adam Palermo ,  Brenda Richards ,  Sergio Pablo Sardi ,  Catherine O′Riordan ,  Antonius Song

    IPC分类号： C07H21/04 ,  C12N15/113

    CPC分类号： C12N15/113 ,  C12N2310/14 ,  C12N2310/531 ,  C12N2310/533

    摘要： Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.