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    TDP-43 knock-in mouse model of amyotrophic lateral sclerosis
    3.
    发明授权
    TDP-43 knock-in mouse model of amyotrophic lateral sclerosis 有权

    公开(公告)号：US11582957B2

    公开(公告)日：2023-02-21

    申请号：US16387430

    申请日：2019-04-17

    Applicant: Academia Sinica

    Inventor： Che-Kun James Shen ,  Shih-Ling Huang

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

    Compounds for uses in pharmacological induction of HBF for treatment of sickle cell disease and ß-thalassemia
    5.
    发明授权
    Compounds for uses in pharmacological induction of HBF for treatment of sickle cell disease and ß-thalassemia 有权

    公开(公告)号：US12246021B2

    公开(公告)日：2025-03-11

    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 ,  A61K31/5377 ,  A61P7/06 ,  C07D473/16 ,  C07D473/18

    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.

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