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    GENE THERAPY FOR SPINAL CORD DISORDERS
    1.
    发明申请
    GENE THERAPY FOR SPINAL CORD DISORDERS 有权

    公开(公告)号：US20210008227A1

    公开(公告)日：2021-01-14

    申请号：US16910834

    申请日：2020-06-24

    Applicant: Genzyme Corporation

    Inventor： James DODGE ,  Lamya S. SHIHABUDDIN ,  Marco A. PASSINI ,  Seng H. CHENG ,  Catherine O'RIORDAN

    IPC: A61K48/00 ,  C12N15/864 ,  C07K14/475

    Abstract: This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention provides a method to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene. The viral vector delivers the transgene to a region of the deep cerebellar nuclei region of the brain. Also provided are compositions and methods to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene to the motor cortex region of the subject's brain.

    GENE THERAPY FOR AMYOTROPHIC LATERAL SCLEROSIS AND OTHER SPINAL CORD DISORDERS
    3.
    发明申请
    GENE THERAPY FOR AMYOTROPHIC LATERAL SCLEROSIS AND OTHER SPINAL CORD DISORDERS 审中-公开
    脊柱侧凸性脊柱炎及其他脊髓损伤的基因治疗

    公开(公告)号：US20160067312A1

    公开(公告)日：2016-03-10

    申请号：US14692469

    申请日：2015-04-21

    Applicant: GENZYME CORPORATION

    Inventor： James DODGE ,  Lamya SHIHABUDDIN ,  Catherine O'RIORDAN

    IPC: A61K38/30

    Abstract: This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain.

    Abstract translation: 本公开提供了用于治疗影响受试者的运动功能和控制的疾病或损伤的方法和组合物。 一方面，本发明通过将包含转基因的重组神经营养病毒载体给予脑，将转基因产物递送至受试者的脊髓。 病毒载体将转基因递送到易受病毒感染并表达编码的重组病毒基因产物的脑区域。 还提供了通过将含有转基因的重组神经营养病毒载体给予受试者的脑，将转基因产物递送至受试者的脊髓的组合物。

    GENE THERAPY FOR LYSOSOMAL STORAGE DISEASES
    5.
    发明申请
    GENE THERAPY FOR LYSOSOMAL STORAGE DISEASES 审中-公开
    遗传物质储存疾病的基因治疗

    公开(公告)号：US20150151007A1

    公开(公告)日：2015-06-04

    申请号：US14328621

    申请日：2014-07-10

    Applicant: Genzyme Corporation

    Inventor： James DODGE ,  Seng CHENG

    IPC: A61K48/00 ,  A61K38/47

    CPC classification number: A61K48/0075 ,  A61K38/47 ,  C12N15/86 ,  C12N2750/14143 ,  C12Y302/01045

    Abstract: This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.

    Abstract translation: 本公开提供了用于治疗受试者中溶酶体贮积病的方法和组合物。 在本发明的一个方面，通过将包含转基因的重组神经营养病毒载体给予脑，将转基因产物递送至受试者。 病毒载体将转基因递送到易受病毒感染并表达编码的重组病毒基因产物的脑区域。 还提供了通过将含有转基因的重组神经营养病毒载体给予受试者的脑，将转基因产物递送给受试者的组合物。 转基因产物可以是溶酶体贮积病中缺乏的任何一种。

    GENE THERAPY FOR NEUROMETABOLIC DISORDERS
    7.
    发明申请
    GENE THERAPY FOR NEUROMETABOLIC DISORDERS 审中-公开

    公开(公告)号：US20200306387A1

    公开(公告)日：2020-10-01

    申请号：US16808206

    申请日：2020-03-03

    Applicant: Genzyme Corporation

    Inventor： Marco A. PASSINI ,  James DODGE

    IPC: A61K48/00 ,  C12N15/86 ,  C12N15/864 ,  C07K14/47

    Abstract: The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.

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