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    Methods and kits for diagnosing, prognosing and monitoring parkinson's disease
    31.
    发明授权
    Methods and kits for diagnosing, prognosing and monitoring parkinson's disease 有权

    公开(公告)号：US09970056B2

    公开(公告)日：2018-05-15

    申请号：US14870960

    申请日：2015-09-30

    Applicant: Rosalind Franklin University of Medicine and Science

    Inventor： Judith Ann Potashkin ,  Jose Alfredo Santiago

    IPC: C12Q1/68

    CPC classification number: C12Q1/6883 ,  C12Q2600/158

    Abstract: Network-based meta-analysis of four independent microarray studies identified the hepatocyte nuclear factor (HNF4A), a transcription factor associated with gluconeogenesis and diabetes, as a central regulatory hub gene upregulated in blood of PD patients. In parallel, the polypyrimidine tract binding protein 1 (PTBP1), involved in the stabilization and mRNA translation of insulin, was identified as the most downregulated gene. Using both markers, PD patients were classified with 90% sensitivity and 80% specificity. Longitudinal performance analysis demonstrated that relative abundance of HNF4A and PTBP1 mRNAs significantly decreased and increased, respectively, in PD patients during 3 years follow up period. The inverse regulation of HNF4A and PTBP1 provides a molecular rationale for the altered insulin signaling observed in PD patients. The longitudinally dynamic biomarkers identified in this study may be useful for monitoring disease-modifying therapies for PD.

    ANTISENSE COMPOUNDS TARGETING GENES ASSOCIATED WITH CYSTIC FIBROSIS
    32.
    发明申请
    ANTISENSE COMPOUNDS TARGETING GENES ASSOCIATED WITH CYSTIC FIBROSIS 审中-公开

    公开(公告)号：US20180119152A1

    公开(公告)日：2018-05-03

    申请号：US15835995

    申请日：2017-12-08

    Applicant: Rosalind Franklin University of Medicine and Science

    Inventor： Michelle L. HASTINGS

    IPC: C12N15/113

    Abstract: The present disclosure relates generally to compounds comprising oligonucleotides complementary to a cystic fibrosis transmembrane conductance regulator (CFTR) RNA transcript. Certain such compounds are useful for hybridizing to a CFTR RNA transcript, including but not limited to a CFTR RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CFTR transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Cystic Fibrosis.

    Compounds For Stabilizing Ryanodine Receptors From Aberrant Levels Of Calcium Release
    36.
    发明申请
    Compounds For Stabilizing Ryanodine Receptors From Aberrant Levels Of Calcium Release 有权
    用于稳定来自不稳定水平的钙释放的兰诺定受体的化合物

    公开(公告)号：US20160024038A1

    公开(公告)日：2016-01-28

    申请号：US14716138

    申请日：2015-05-19

    Applicant: Rosalind Franklin University of Medicine and Science

    Inventor： Grace E. Stutzmann ,  Russell Dahl ,  Christopher H. Kaiho

    IPC: C07D307/54 ,  C07D307/52 ,  C07D405/12

    CPC classification number: C07D307/54 ,  C07D307/52 ,  C07D405/12 ,  C07D407/12

    Abstract: Disclosed herein are methods and compositions comprising compounds capable of normalizing neuronal calcium dyshomeostasis. Also disclosed are methods comprising these compounds for treating neuronal or neurological disorders, including Alzheimer's disease, Parkinson's disease, Huntington's disease, fronto-temporal dementia, Pick's disease, chronic traumatic encepholopathy, traumatic brain injury, stroke, cerebellar ataxia, multiple sclerosis, Down syndrome, and aging-related CNS disorders.

    Abstract translation: 本文公开了包含能够使神经元钙发育不稳定性归一化的化合物的方法和组合物。 还公开了包括这些化合物用于治疗神经元或神经障碍的方法，包括阿尔茨海默病，帕金森病，亨廷顿舞蹈病，颞叶性痴呆，皮克病，慢性创伤性脑病，创伤性脑损伤，中风，小脑共济失调，多发性硬化症，唐氏综合征 ，和老化相关的中枢神经系统疾病。

    Compounds For Stabilizing Ryanodine Receptors From Aberrant Levels Of Calcium Release
    38.
    发明申请
    Compounds For Stabilizing Ryanodine Receptors From Aberrant Levels Of Calcium Release 有权
    用于稳定来自不稳定水平的钙释放的兰诺定受体的化合物

    公开(公告)号：US20140275112A1

    公开(公告)日：2014-09-18

    申请号：US14213614

    申请日：2014-03-14

    Applicant: Rosalind Franklin University of Medicine and Science

    Inventor： Grace E. Stutzmann ,  Russell Dahl

    IPC: C07D307/52 ,  C07D307/54 ,  C07D407/12 ,  C07D405/12

    CPC classification number: C07D307/52 ,  A61K31/341 ,  A61K31/4166 ,  A61K31/4427 ,  C07D307/54 ,  C07D405/12 ,  C07D407/12

    Abstract: Disclosed herein are methods and compositions comprising compounds capable of normalizing neuronal calcium dyshomeostasis. Also disclosed are methods comprising these compounds for treating neuronal or neurological disorders, including Alzheimer's disease, Parkinson's disease, Huntington's disease, fronto-temporal dementia, Pick's disease, chronic traumatic encepholopathy, traumatic brain injury, stroke, cerebellar ataxia, multiple sclerosis, Down syndrome, and aging-related CNS disorders.

    Abstract translation: 本文公开了包含能够使神经元钙发育不稳定性归一化的化合物的方法和组合物。 还公开了包括这些化合物用于治疗神经元或神经障碍的方法，包括阿尔茨海默病，帕金森病，亨廷顿舞蹈病，颞叶性痴呆，皮克病，慢性创伤性脑病，创伤性脑损伤，中风，小脑共济失调，多发性硬化症，唐氏综合征 ，和老化相关的中枢神经系统疾病。

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