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    COMPOUNDS FOR USES IN PHARMACOLOGICAL INDUCTION OF HBF FOR TREATMENT OF SICKLE CELL DISEASE AND ß-THALASSEMIA
    2.
    发明申请
    COMPOUNDS FOR USES IN PHARMACOLOGICAL INDUCTION OF HBF FOR TREATMENT OF SICKLE CELL DISEASE AND ß-THALASSEMIA 有权

    公开(公告)号：US20220152036A1

    公开(公告)日：2022-05-19

    申请号：US17439630

    申请日：2020-03-25

    Applicant: Academia Sinica ,  National Health Research Institutes

    Inventor： Weir-Torn JIAANG ,  Che-Kun James SHEN

    IPC: A61K31/52 ,  A61P7/06 ,  C07D473/16 ,  C07D473/18 ,  A61K31/5377

    Abstract: Provided herein are compounds of Formula (I). The compounds described herein are useful in treating a disease associated with the expression of endogenous embryonic/fetal globin (e.g., γ globin) in erythrocytes (e.g., treating β-thalassemia and/or sickle cell disease through at least the induction of the globin gene expression in erythrocytes), and/or anemia (β-thalassemia and/or sickle cell anemia). The compounds described herein are useful in treating, delaying, and/or preventing the adverse effects of β-thalassemia and/or sickle cell disease, inducing γ globin production, and/or inducing the expression of embryonic/fetal globin genes in a subject, cell, tissue, or biological sample. Also provided in the present disclosure are pharmaceutical compositions, kits, and methods of using the compounds for inducing γ globin production described herein and for treating any of the target diseases described herein.

    TDP-43 KNOCK-IN MOUSE MODEL OF AMYOTROPHIC LATERAL SCLEROSIS
    3.
    发明申请
    TDP-43 KNOCK-IN MOUSE MODEL OF AMYOTROPHIC LATERAL SCLEROSIS 审中-公开

    公开(公告)号：US20190335727A1

    公开(公告)日：2019-11-07

    申请号：US16387430

    申请日：2019-04-17

    Applicant: Academia Sinica

    Inventor： Che-Kun James SHEN ,  Shih-Ling HUANG

    IPC: A01K67/027 ,  C07K14/47 ,  C12N5/0793 ,  G01N33/564 ,  A61K49/00 ,  G01N33/50

    Abstract: The invention relates to a genetically modified mouse comprising a heterozygous mutation of Tardbp (TDP-43) gene in that the Asn at amino acid 390 in TDP-43 is substituted with an amino acid that is different from Asn, wherein the genetically modified mouse exhibits Amyotrophic lateral sclerosis (ALS)-like phenotypes, TDP-43 proteinopathies and/or motor neuron degeneration. The invention also so relates to an isolated spinal cord motor neuron differentiated from an embryonic stem cell (ESC) that is obtained from an offspring of a genetically modified mouse according to the invention. Methods for identifying an agent alleviating and/or suppressing ALS-TDP pathogenesis are also disclosed.

    DNA 5-METHYL CYTOSINE DEMETHYLATION ACTIVITY OF VERTEBRATE DNA METHYLTRANSFERASES
    7.
    发明申请
    DNA 5-METHYL CYTOSINE DEMETHYLATION ACTIVITY OF VERTEBRATE DNA METHYLTRANSFERASES 有权
    DNA甲基衍生物的DNA 5-甲基胞嘧啶脱乙酰壳多糖活性

    公开(公告)号：US20150315628A1

    公开(公告)日：2015-11-05

    申请号：US14647816

    申请日：2013-12-10

    Applicant: ACADEMIA SINICA

    Inventor： Che-Kun James SHEN ,  Chun-Chang CHEN

    IPC: C12Q1/48 ,  C12Q1/68

    CPC classification number: C12Q1/48 ,  C12Q1/68 ,  C12Q1/6897 ,  G01N2333/91011 ,  G01N2500/04 ,  C12Q2521/331 ,  C12Q2525/143

    Abstract: Methods for identifying a test agent as a modulator of the active DNA demethylation activity of a DNA methyltransferase are disclosed. The method comprises: a) providing a methylated DNA; b) providing the DNA methyltransferase; c) allowing the methylated DNA to react with the DNA methyltransferase for a sufficient time to perform a demethylation reaction and generate a demethylated DNA product in the presence or absence of a test agent; d) analyzing the extent of demethylation; and d) comparing the extents of the demethylation in the presence and absence of the test agent, and thereby identify the test agent as a modulator of the DNA demethylation activity of the DNA methyltransferase.

    Abstract translation: 公开了用于鉴定测试剂作为DNA甲基转移酶的活性DNA去甲基化活性的调节剂的方法。 该方法包括：a）提供甲基化DNA; b）提供DNA甲基转移酶; c）允许甲基化DNA与DNA甲基转移酶反应足够的时间进行去甲基化反应，并在试剂存在或不存在的情况下产生去甲基化的DNA产物; d）分析去甲基化程度; 和d）比较测试试剂存在和不存在时的去甲基化程度，从而鉴定作为DNA甲基转移酶的DNA去甲基化活性的调节剂的测试试剂。

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