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    METHODS FOR ENHANCING LIFESPAN AND/OR TREATING CELLULAR PROLIFERATIVE DISORDERS BY TRANSPLANTATION
    1.
    发明申请
    METHODS FOR ENHANCING LIFESPAN AND/OR TREATING CELLULAR PROLIFERATIVE DISORDERS BY TRANSPLANTATION 审中-公开
    通过移植增强寿命和/或治疗细胞增殖性疾病的方法

    公开(公告)号：WO2018052964A1

    公开(公告)日：2018-03-22

    申请号：PCT/US2017/051310

    申请日：2017-09-13

    Applicant: ACADEMIA SINICA ,  SHEN, Che-Kun James ,  SHYU, Yu-Chiau ,  HUNG, Chun-Hao

    Inventor： SHEN, Che-Kun James ,  SHYU, Yu-Chiau ,  HUNG, Chun-Hao

    IPC: A61K38/16 ,  C07K14/47 ,  C12N5/095

    Abstract: The invention found that first, the feasibility of transfer of tumor resistance and other healthy longevity characters through transplantation of bone marrow mononuclear cells (BMMNC) or hematopoietic stem cells (HSC)/hematopoietic stem and progenitor cells (HSPC) consisting of genetically engineered EKLF gene encoding the hematopoietic transcription factor EKLF. Secondly, the present invention demonstrates expression of EKLF in the long-term hematopoietic stem cells (LT-HSC), and thus EKLF as a target of regulation of hematopoiesis.

    Abstract translation: 本发明首先通过移植骨髓单个核细胞（BMMNC）或造血干细胞（HSC）/造血干细胞和祖细胞（以下简称“造血干细胞”）转移肿瘤抗性和其他健康长寿特征 HSPC）由编码造血转录因子EKLF的基因工程EKLF基因组成。 其次，本发明展示了EKLF在长期造血干细胞（LT-HSC）中的表达，并且因此表明EKLF作为造血调节的靶标。

    COMPOUNDS FOR USES IN PHARMACOLOGICAL INDUCTION OF HBF FOR TREATMENT OF SICKLE CELL DISEASE AND β-THALASSEMIA
    3.
    发明申请
    COMPOUNDS FOR USES IN PHARMACOLOGICAL INDUCTION OF HBF FOR TREATMENT OF SICKLE CELL DISEASE AND β-THALASSEMIA 审中-公开

    公开(公告)号：WO2020198367A1

    公开(公告)日：2020-10-01

    申请号：PCT/US2020/024727

    申请日：2020-03-25

    Applicant: ACADEMIA SINICA ,  NATIONAL HEALTH RESEARCH INSTITUTES

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

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

    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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