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    METHODS FOR TREATING MUSCULAR DYSTROPHY USING INHIBITORY OLIGONUCLEOTIDES TO CD49D
    1.
    发明申请
    METHODS FOR TREATING MUSCULAR DYSTROPHY USING INHIBITORY OLIGONUCLEOTIDES TO CD49D 有权

    公开(公告)号：US20220296631A1

    公开(公告)日：2022-09-22

    申请号：US17609334

    申请日：2020-05-06

    申请人： Antisense Therapeutics Ltd

    发明人： George Tachas

    IPC分类号： A61K31/7115 ,  A61K31/573 ,  A61K31/58 ,  A61K31/7125 ,  A61K45/06 ,  A61P21/00 ,  C12N15/113

    摘要： A method of modifying muscle or limb performance in a subject with or at risk of a condition associated with muscle atrophy, muscle fatty tissue, or pseudohypertrophy or a muscular dystrophy, by administering a pharmaceutical composition an inhibitory oligonucleotide to CD49d sufficient to modify one or more markers, signs or parameters of muscle fat, muscle performance or function, or limb performance or function. A method comprising the following steps: (i) determining the level of CD4+CD49d+ T cells in a blood sample from the subject; (ii) administering a course of antisense oligonucleotide and repeating step (i) at least once towards the end of the dosing period; (iii) within one week of dose completion repeat step (i); (iv) processing the results to determine whether the subject has or has not displayed a post-dose completion rebound, stability or loss in the level of CD4+CD49d+ T cells.

    Mobilizing leukemia cells
    9.
    发明授权
    Mobilizing leukemia cells 有权

    公开(公告)号：US11041156B2

    公开(公告)日：2021-06-22

    申请号：US15971938

    申请日：2018-05-04

    申请人： Antisense Therapeutics Ltd ,  Children's Hospital of Los Angeles

    发明人： George Tachas ,  Yong-Mi Kim

    IPC分类号： C07H21/04 ,  C12N15/113 ,  A61K31/7125 ,  A61K45/06 ,  A61P35/02 ,  A61K31/7076

    摘要： A method for mobilizing leukemia cells which are α4 integrin positive to the peripheral blood of a human subject, the method comprising administering to the human subject an effective amount of an antisense compound to α4 integrin. The cells may be mobilized from bone marrow. The antisense compound is: 5′-MeCMeUG AGT MeCTG TTT MeUMeCMeC AMeUMeU MeCMeU-3′ wherein, (a) each of the 19 internucleotide linkages of the oligonucleotide is an O,O-linked phosphorothioate diester; (b) the nucleotides at the positions 1 to 3 from the 5′ end are 2′-O-(2-methoxyethyl) modified ribonucleosides; (c) the nucleotides at the positions 4 to 12 from the 5′ end are 2′-deoxyribonucleosides; (d) the nucleotides at the positions 13 to 20 from the 5′ end are 2′-O-(2-methoxyethyl) modified ribonucleosides; and (e) all cytosines are 5-methylcytosines (MeC), or a pharmaceutically acceptable salt thereof.