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    DOUBLE-STRANDED/SELF-COMPLEMENTARY VECTORS WITH A TRUNCATED CBA PROMOTER AND METHODS OF GENE DELIVERY
    2.
    发明申请
    DOUBLE-STRANDED/SELF-COMPLEMENTARY VECTORS WITH A TRUNCATED CBA PROMOTER AND METHODS OF GENE DELIVERY 有权
    具有截断CBA促进器的双重条纹/自补偿矢量和基因交付方法

    公开(公告)号：US20100069467A1

    公开(公告)日：2010-03-18

    申请号：US12298845

    申请日：2007-04-27

    申请人： Sanford L. Boye ,  William W. Hauswirth ,  Barry Jonh Byrne

    发明人： Sanford L. Boye ,  William W. Hauswirth ,  Barry Jonh Byrne

    IPC分类号： A61K31/7088 ,  C12N15/63 ,  C12N5/00 ,  C12N5/071

    CPC分类号： C12N15/86 ,  C12N2750/14143 ,  C12N2830/15

    摘要： The present inventors concerns vectors carrying a truncated chimeric CMV-chicken β-actin (smCBA) promoter in which the hybrid chicken β-actin/rabbit β-globin intron is greatly shortened, and their use to deliver to an operatively linked polynucleotide to host cells in vitro or in vivo, resulting in expression of the polynucleotide in the host cells. In one embodiment, the vector carrying the smCBA promoter is administered to the eye. In another embodiment, the vector carrying the smCBA promoter is a self-complementary adeno-associated virus (AAV). The AAV vector may be of any serotype (e.g., type 1, type 2, type 3, type 4, type 5, type 6, type 7, type 8, type 9, type 10). In another embodiment, a self-complementary vector carrying the smCBA promoter is administered to the eye. Another aspect of the invention concerns host cells carrying a vector of the invention. Another aspect of the invention concerns pharmaceutical composition comprising the vectors or host cells of the invention, and a pharmaceutically acceptable carrier.

    摘要翻译： 本发明人涉及携带截短的嵌合CMV-鸡＆bgr-肌动蛋白（smCBA）启动子的载体，其中杂交的鸡和哺乳动物/兔和珠蛋白内含子大大缩短，并且它们用于递送至可操作地连接的多核苷酸 在体外或体内宿主细胞，导致多核苷酸在宿主细胞中的表达。 在一个实施方案中，携带smCBA启动子的载体被施用于眼睛。 在另一个实施方案中，携带smCBA启动子的载体是自身互补的腺相关病毒（AAV）。 AAV载体可以是任何血清型（例如1型，2型，3型，4型，5型，6型，7型，8型，9型，10型）。 在另一个实施方案中，将携带smCBA启动子的自身互补载体施用于眼睛。 本发明的另一方面涉及携带本发明载体的宿主细胞。 本发明的另一方面涉及包含本发明的载体或宿主细胞的药物组合物和药学上可接受的载体。

    rAAV-Guanylate Cyclase Compositions and Methods for Treating Lebers Congenital Amaurosis-1 (LCA1)
    6.
    发明申请
    rAAV-Guanylate Cyclase Compositions and Methods for Treating Lebers Congenital Amaurosis-1 (LCA1) 有权
    rAAV-Guanylate Cyclase组合物和治疗Lebers先天性黑霉素1（LCA1）的方法

    公开(公告)号：US20130210895A1

    公开(公告)日：2013-08-15

    申请号：US13643074

    申请日：2011-04-22

    申请人： Shannon Elizabeth Boye ,  William W. Hauswirth ,  Sanford Leon Boye

    发明人： Shannon Elizabeth Boye ,  William W. Hauswirth ,  Sanford Leon Boye

    IPC分类号： C12N15/861

    CPC分类号： C12N15/861 ,  A61K38/00 ,  A61K48/005 ,  C12N9/88 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/85 ,  C12Y406/01002

    摘要： Disclosed are viral vector compositions comprising polynucleotide sequences that express one or more biologically-active mammalian guanylate cyclase proteins. Also disclosed are methods for their use in preventing, treating, and/or ameliorating at least one or more symptoms of a disease, disorder, abnormal condition, or dysfunction resulting at least in part from a guanylate cyclase deficiency in vivo. In particular embodiments, the use of recombinant adeno-associated viral (rAAV) vectors to treat or ameliorate symptoms of Leber's congenital amaurosis, as well as other conditions caused by an absence or reduction in the expression of a functional retinal-specific guanylate cyclase 1 (retGC1).

    摘要翻译： 公开了包含表达一种或多种生物活性哺乳动物鸟苷酸环化酶蛋白的多核苷酸序列的病毒载体组合物。 还公开了它们用于预防，治疗和/或改善至少部分由体内鸟苷酸环化酶缺陷导致的疾病，病症，异常状况或功能障碍的症状的方法。 在具体实施方案中，使用重组腺相关病毒（rAAV）载体来治疗或改善Leber先天性黑斑症的症状，以及由功能性视网膜特异性鸟苷酸环化酶1的表达的不存在或减少引起的其它病症（ retGC1）。