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    REPROGRAMMING OF HUMAN WHARTONS JELLY CELLS TO PRODUCE HAIR CELLS
    2.
    发明申请
    REPROGRAMMING OF HUMAN WHARTONS JELLY CELLS TO PRODUCE HAIR CELLS 有权
    人乳头瘤细胞生成头发细胞

    公开(公告)号：US20150307837A1

    公开(公告)日：2015-10-29

    申请号：US14698157

    申请日：2015-04-28

    申请人： Adam J. Mellott ,  Michael Detamore ,  Hinrich Staecker

    发明人： Adam J. Mellott ,  Michael Detamore ,  Hinrich Staecker

    IPC分类号： C12N5/0793 ,  C12N15/113 ,  A61K35/30 ,  C07K14/47

    CPC分类号： C12N5/062 ,  A61K35/30 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/51 ,  C12N2506/025 ,  C12N2510/00 ,  C12N2533/90

    摘要： A method of transforming human cells into mechanosensory hair cells (MHCs), such as inner hear hair cells in the cochlea and vestibular organs, can include: causing human Wharton's jelly cells (hWJCs) to increase expression of or biological function of HATH1 so as to transform the hWJCs into MHCs. The method can include; administering a nucleic acid that encodes HATH1 to the hWJCs; causing inhibited expression of or biological function of HES1 and/or HES5 in the hWJCs; administering a nucleic acid that inhibits HES1 and/or a nucleic acid that inhibits HES5 to the hWJCs; causing inhibited expression of or biological function of HES1 and/or HES5 in the WJCs by administering a nucleic acid that inhibits HES1 and/or a nucleic acid that inhibits HES5; nucleic acids are administered includes a sequence of SEQ ID NO: 2, SEQ ID NO: 3, and/or SEQ ID NO: 4.

    摘要翻译： 将人类细胞转化为机械感染毛细胞（MHC）的方法，例如耳蜗和前庭器官内部听到的毛细胞，可以包括：引起人类沃顿的果冻细胞（hWJCs）增加HATH1的表达或生物学功能，以便 将hWJCs转化为MHC。 该方法可以包括 向hWJCs施用编码HATH1的核酸; 导致hWJCs中HES1和/或HES5的表达或生物学功能受到抑制; 施用抑制HES1的核酸和/或对hWJC抑制HES5的核酸; 通过施用抑制HES1的核酸和/或抑制HES5的核酸来抑制WJCs中HES1和/或HES5的表达或生物学功能; 核酸被给予包括SEQ ID NO：2，SEQ ID NO：3和/或SEQ ID NO：4的序列。

    Reprogramming of human whartons jelly cells to produce hair cells
    3.
    发明授权
    Reprogramming of human whartons jelly cells to produce hair cells 有权
    重新编程人类whartons果冻细胞产生毛细胞

    公开(公告)号：US09512400B2

    公开(公告)日：2016-12-06

    申请号：US14698157

    申请日：2015-04-28

    申请人： Adam J. Mellott ,  Michael Detamore ,  Hinrich Staecker

    发明人： Adam J. Mellott ,  Michael Detamore ,  Hinrich Staecker

    IPC分类号： A61K35/30 ,  C12N15/113 ,  C12N5/0793

    CPC分类号： C12N5/062 ,  A61K35/30 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/51 ,  C12N2506/025 ,  C12N2510/00 ,  C12N2533/90

    摘要： A method of transforming human cells into mechanosensory hair cells (MHCs), such as inner hear hair cells in the cochlea and vestibular organs, can include: causing human Wharton's jelly cells (hWJCs) to increase expression of or biological function of HATH1 so as to transform the hWJCs into MHCs. The method can include; administering a nucleic acid that encodes HATH1 to the hWJCs; causing inhibited expression of or biological function of HES1 and/or HES5 in the hWJCs; administering a nucleic acid that inhibits HES1 and/or a nucleic acid that inhibits HES5 to the hWJCs; causing inhibited expression of or biological function of HES1 and/or HES5 in the WJCs by administering a nucleic acid that inhibits HES1 and/or a nucleic acid that inhibits HES5; nucleic acids are administered includes a sequence of SEQ ID NO: 2, SEQ ID NO: 3, and/or SEQ ID NO: 4.

    摘要翻译： 将人类细胞转化为机械感染毛细胞（MHC）的方法，例如耳蜗和前庭器官内部听到的毛细胞，可以包括：引起人类沃顿的果冻细胞（hWJCs）增加HATH1的表达或生物学功能，以便 将hWJCs转化为MHC。 该方法可以包括 向hWJCs施用编码HATH1的核酸; 导致hWJCs中HES1和/或HES5的表达或生物学功能受到抑制; 施用抑制HES1的核酸和/或对hWJC抑制HES5的核酸; 通过施用抑制HES1的核酸和/或抑制HES5的核酸来抑制WJCs中HES1和/或HES5的表达或生物学功能; 核酸被给予包括SEQ ID NO：2，SEQ ID NO：3和/或SEQ ID NO：4的序列。

    PEPTIDE TARGETING OF INNER EAR CELLS
    4.
    发明申请
    PEPTIDE TARGETING OF INNER EAR CELLS 审中-公开
    内皮细胞的肽靶向

    公开(公告)号：US20130096070A1

    公开(公告)日：2013-04-18

    申请号：US13263719

    申请日：2010-04-06

    申请人： Hinrich Staecker ,  Peter Gochee

    发明人： Hinrich Staecker ,  Peter Gochee

    IPC分类号： A61K38/08 ,  C07K7/06

    CPC分类号： A61K38/08 ,  A61K38/00 ,  C07K7/06

    摘要： Peptide targeting moieties that target the cells of the inner ear can be used for targeted therapeutics. As such, nucleic acids and/or drugs can be associated with the targeting moieties in order to provide therapeutics that are delivered to specific cells in the inner ear. Conjugation of drugs or gene therapy vectors to cell specific peptides may allow the treatment of individual cell types within the inner ear. The peptide targeting moieties can be polypeptides having the sequences of Table 1 in an unnatural configuration. The polynucleotide can either consist of the sequence or include additional polypeptides attached to the ends of the sequences shown in Table 1. The polynucleotide can be in a non-native configuration. For example, the polypeptide is selected from the following: a-h-p-h-h-s-m (SEQ ID NO: 12); h-p-h-h-r-i-f (SEQ ID NO: 29); t-v-p-q-l-t-t (SEQ ID NO: 1); s-t-t-k-l-a-l (SEQ ID NO: 2); m-e-g-y-i-h-r (SEQ ID NO: 3); h-a-i-y-p-r-h (SEQ ID NO: 5); h-s-r-l-l-d-q (SEQ ID NO: 6); i-q-s-p-h-f-f (SEQ ID NO: 7); or y-a-a-h-r-s-h (SEQ ID NO: 8).

    摘要翻译： 靶向内耳细胞的肽靶向部分可用于靶向治疗。 因此，核酸和/或药物可以与靶向部分相关联，以提供递送至内耳中的特定细胞的治疗剂。 药物或基因治疗载体与细胞特异性肽的缀合可以允许治疗内耳内的各种细胞类型。 肽靶向部分可以是具有表1序列的非天然构型的多肽。 多核苷酸可以由序列组成或包括连接到表1所示序列末端的其他多肽。多核苷酸可以是非天然构型。 例如，多肽选自如下：a-h-p-h-h-s-m（SEQ ID NO：12）; h-p-h-h-r-i-f（SEQ ID NO：29）; t-v-p-q-1-t-t（SEQ ID NO：1）; s-t-t-k-1-a-1（SEQ ID NO：2）; m-e-g-y-i-h-r（SEQ ID NO：3）; h-a-i-y-p-r-h（SEQ ID NO：5）; h-s-r-1-d-q（SEQ ID NO：6）; i-q-s-p-h-f-f（SEQ ID NO：7）; 或y-a-a-h-r-s-h（SEQ ID NO：8）。