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    COMPOUNDS AND METHODS FOR MODULATING CLN3 EXPRESSION
    11.
    发明申请
    COMPOUNDS AND METHODS FOR MODULATING CLN3 EXPRESSION 有权

    公开(公告)号：US20220280545A1

    公开(公告)日：2022-09-08

    申请号：US17274981

    申请日：2019-09-10

    Applicant: Ionis Pharmaceuticals, Inc. ,  Rosalind Franklin University of Medicine & Science

    Inventor： Michelle L. Hastings ,  Frank Rigo

    IPC: A61K31/712 ,  A61K31/7125 ,  A61K31/713 ,  A61K47/02

    Abstract: Provided are compounds, methods, and pharmaceutical compositions for modulating the expression of CLN3 RNA in a cell or animal, and in certain instances modulating the expression of CLN3 protein in a cell or animal Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such N symptoms and hallmarks include poor motor function, seizures, vision loss, poor cognitive function, psychiatric problems, accumulation of autofluorescent ceroid lipopigment, brain tissue dysfunction or cell death, accumulation of mitochondrial ATP synthase subunit C, accumulation of lipofuscin, or astrocyte activation in brain tissue.

    Antisense compounds targeting genes associated with cystic fibrosis
    14.
    发明授权
    Antisense compounds targeting genes associated with cystic fibrosis 有权

    公开(公告)号：US11116785B2

    公开(公告)日：2021-09-14

    申请号：US16730517

    申请日：2019-12-30

    Applicant: Rosalind Franklin University of Medicine and Science

    Inventor： Michelle L. Hastings

    IPC: A61K31/712 ,  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.

    Intelligent Offloading Insole Device
    17.
    发明申请
    Intelligent Offloading Insole Device 审中-公开

    公开(公告)号：US20180132566A1

    公开(公告)日：2018-05-17

    申请号：US15813613

    申请日：2017-11-15

    Applicant: ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE ,  THE BOARD OF TRUSTEES OF THE UNIVERSITY OF ILLINOIS

    Inventor： Noah J. Rosenblatt ,  Ryan Crews ,  Farid Amirouche

    IPC: A43B17/03 ,  A43B3/00 ,  A43B17/02 ,  G05B15/02

    Abstract: A plantar surface pressure offloading system includes an insole capable of coupling with a shoe and interfacing with a foot. A number of compressible bladders and pressure sensors are coupled to the insole. Each bladder has an adjustable compressibility, and each pressure sensor is configured to measure a pressure exerted on a respective portion of the foot. A controller of the system can perform, for each compressible bladder, a compressibility adjustment process including (i) receiving, from a respective pressure sensor associated with a respective bladder, a signal indicative of a pressure exerted on a respective portion of the foot, (ii) determining, based on the signal, that the pressure exerted on the respective portion of the foot exceeds a threshold pressure, and (iii) responsive to the determination, adjusting the compressibility of the respective bladder, thereby offloading pressure from the respective portion of the foot to a different portion of the foot.

    Foot-strength test device and methods for use
    18.
    发明授权
    Foot-strength test device and methods for use 有权

    公开(公告)号：US09839389B2

    公开(公告)日：2017-12-12

    申请号：US14689550

    申请日：2015-04-17

    Applicant: Rosalind Franklin University of Medicine and Science

    Inventor： Karen Marie Stevens

    IPC: A61B5/00 ,  A61B5/22

    CPC classification number: A61B5/224

    Abstract: The present disclosure provides a device for testing the strength of a human foot. The device includes a substantially planar member having a top surface and a bottom surface. The device also includes a foot-engaging member moveable along the top surface substantially planar member from a first position to a second position. In addition, the device includes a force sensor fixed with respect to the substantially planar member, where the force sensor resists movement between the first position and the second position. Further, the device includes a tension bearing element connecting the foot-engaging member to the force sensor.

    METHODS AND KITS FOR DIAGNOSING, PROGNOSING AND MONITORING PARKINSON'S DISEASE
    20.
    发明申请
    METHODS AND KITS FOR DIAGNOSING, PROGNOSING AND MONITORING PARKINSON'S DISEASE 有权
    诊断，预防和监测帕金森病的方法和工具

    公开(公告)号：US20160244833A1

    公开(公告)日：2016-08-25

    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.

    Abstract translation: 四个独立的微阵列研究的基于网络的荟萃分析确定了肝细胞核因子（HNF4A），一种与糖异生和糖尿病相关的转录因子，作为PD患者血液中枢调节中枢基因。 同时，涉及胰岛素的稳定和mRNA翻译的聚嘧啶多肽结合蛋白1（PTBP1）被鉴定为最下调的基因。 使用两个标记物，PD患者被分类为90％敏感性和80％特异性。 纵向性能分析表明，3年随访期间，PD患者HNF4A和PTBP1 mRNA的相对丰度分别显着降低和升高。 HNF4A和PTBP1的逆调节为PD患者观察到的改变的胰岛素信号提供了分子依据。 在本研究中鉴定的纵向动态生物标志物可能用于监测PD的疾病修复治疗。

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