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    CAPSID-FREE AAV VECTORS, COMPOSITIONS, AND METHODS FOR VECTOR PRODUCTION AND GENE DELIVERY
    2.
    发明申请
    CAPSID-FREE AAV VECTORS, COMPOSITIONS, AND METHODS FOR VECTOR PRODUCTION AND GENE DELIVERY 有权
    不含CAPSID的AAV矢量，组合物和方法用于矢量生产和基因分送

    公开(公告)号：US20140107186A1

    公开(公告)日：2014-04-17

    申请号：US14004379

    申请日：2012-03-12

    Applicant: Luis Garcia ,  Cyriaque Beley ,  Thomas Voit ,  Robert Michael Kotin

    Inventor： Luis Garcia ,  Cyriaque Beley ,  Thomas Voit ,  Robert Michael Kotin

    IPC: C12N15/86

    CPC classification number: C12N15/86 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2800/24

    Abstract: An isolated linear nucleic acid molecule comprising in this order: a first adeno-associated virus (AAV) inverted terminal repeat (ITR), a nucleotide sequence of interest and a second AAV ITR, wherein said nucleic acid molecule is devoid of AAV capsid protein coding sequences. The said nucleic acid molecule can be applied to a host at repetition without eliciting an immune response. Methods for producing and purifying this nucleic acid molecule, and use of the same for therapeutic purposes are also provided.

    Abstract translation: 一种分离的线性核酸分子，其以以下顺序包含：第一腺相关病毒（AAV）倒置末端重复（ITR），目的核苷酸序列和第二AAV ITR，其中所述核酸分子缺乏AAV衣壳蛋白编码 序列。 所述核酸分子可以重复施用于宿主而不引发免疫应答。 还提供了用于产生和纯化该核酸分子的方法及其用于治疗目的的用途。

    Apparatus for delivering pipettable substances
    4.
    发明授权
    Apparatus for delivering pipettable substances 有权
    用于输送可移液物质的装置

    公开(公告)号：US08440151B2

    公开(公告)日：2013-05-14

    申请号：US12673755

    申请日：2008-08-18

    Applicant: Thomas Voit

    Inventor： Thomas Voit

    IPC: B01L3/00

    CPC classification number: B65B69/0041 ,  B01L3/50853 ,  B01L2200/16 ,  B01L2300/044 ,  B01L2300/0672 ,  B01L2300/0832 ,  B65D77/08 ,  G01N35/1079

    Abstract: A system (10) for removing a pipettable substance from a pre-filled container (20), which is closed off by a top (30) having at least one opening area (40), comprises an opening tool (100) having a tube (110), which has a cross-section corresponding substantially to the shape of the opening area and which comprises at a distal end (120) an endpiece (140) extending substantially obliquely relative to the longitudinal axis of the tube, which moves a part of the top (30) located inside the opening area (40) towards the container when the opening tool is applied, so as to form an opening in the top, and a point of attack (150) for a transporting tool (200). The opening tool (100) is designed to remain on the container (20) after use. Moreover, the system comprises a transporting tool (200) for moving the opening tool (100) through the lid, and a cannula (250), which is adapted to be inserted over at least part of its length through the tube (110) into the interior of the container (20), in the position of use of the opening tool (100), and one end of which can be connected to a suction device (300) for sucking a pipettable substance out of the interior of the container (20).

    Abstract translation: 一种用于从预填充容器（20）中移除可移液物质的系统（10），其由具有至少一个开口区域（40）的顶部（30）封闭，包括具有管子的开口工具（100） （110），其横截面基本上对应于开口区域的形状，并且在远端（120）处包括相对于管的纵向轴线基本上倾斜地延伸的端部件（140），所述端件（110）移动部件 当打开工具被施加时，位于开口区域（40）内部的顶部（30）朝向容器移动，以便在顶部形成开口以及用于输送工具（200）的攻角（150）。 打开工具（100）设计成在使用后留在容器（20）上。 此外，该系统包括用于使打开工具（100）通过盖移动的输送工具（200）和套管（250），套管（250）适于在其长度的至少一部分上穿过管（110）插入， 所述容器（20）的内部处于使用所述打开工具（100）的位置，并且其一端可以连接到用于从所述容器的内部吸出可移液物质的抽吸装置（300） 20）。

    TRICYCLO-DNA ANTISENSE OLIGONUCLEOTIDES, COMPOSITIONS, AND METHODS FOR THE TREATMENT OF DISEASE
    6.
    发明申请
    TRICYCLO-DNA ANTISENSE OLIGONUCLEOTIDES, COMPOSITIONS, AND METHODS FOR THE TREATMENT OF DISEASE 审中-公开
    TRICYCLO-DNA抗体寡核苷酸，组合物和治疗疾病的方法

    公开(公告)号：US20120149756A1

    公开(公告)日：2012-06-14

    申请号：US13263949

    申请日：2010-04-09

    Applicant: Daniel Schümperli ,  Christian Leumann ,  Denis Furling ,  Luis Garcia ,  Thomas Voit

    Inventor： Daniel Schümperli ,  Christian Leumann ,  Denis Furling ,  Luis Garcia ,  Thomas Voit

    IPC: A61K31/712 ,  A61P21/00 ,  A61P25/00 ,  C07H21/04 ,  C12N5/071

    CPC classification number: C12N15/113 ,  A61K31/711 ,  A61K31/712 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/3231 ,  C12N2320/33

    Abstract: Provided are tricyclo-DNA (tc-DNA) AON and methods employing tc-DNA AON for modifying splicing events that occur during pre-mRNA processing. Tricyclo-DNA (tc-DNA) AON are described that may be used to facilitate exon skipping or to mask intronic silencer sequences and/or terminal stem-loop sequences during pre-mRNA processing and to target RNase-mediated destruction of processed mRNA. Tc-DNA AON described herein may be used in methods for the treatment of Duchenne Muscular Dystrophy by skipping a mutated exon 23 or exon 51 within a dystrophin gene to restore functionality of a dystrophin protein; in methods for the treatment of Spinal Muscular Atrophy by masking an intronic silencing sequence and/or a terminal stem-loop sequence within an SMN2 gene to yield modified functional SMN2 protein, including an amino acid sequence encoded by exon 7, which is capable of at least partially complementing a non-functional SMN1 protein; and in methods for the treatment of Steinert's Myotonic Dystrophy by targeting the destruction of a mutated DM1 mRNA comprising 3′-terminal CUG repeats.

    Abstract translation: 提供三环DNA（tc-DNA）AON和使用tc-DNA AON来改变在mRNA前处理期间发生的剪接事件的方法。 描述了Tricyclo-DNA（tc-DNA）AON，其可用于促进外显子跳跃或在mRNA前处理期间掩盖内含子沉默子序列和/或末端茎环序列，并靶向核糖核酸酶介导的加工mRNA的破坏。 本文所述的Tc-DNA AON可用于通过跳过肌营养不良蛋白基因内突变外显子23或外显子51来恢复肌营养不良蛋白功能的方法来治疗杜氏肌营养不良症; 在通过掩蔽SMN2基因内的内含子沉默序列和/或末端茎环序列来治疗脊髓性肌萎缩的方法中，以产生修饰的功能性SMN2蛋白，包括由外显子7编码的氨基酸序列，其能够在 最少部分补充非功能性SMN1蛋白; 以及用于通过靶向包含3'-末端CUG重复序列的突变的DM1mRNA的破坏来治疗Steinert's强直性营养不良的方法。

    SAMPLE RECEPTACLE
    10.
    发明申请
    SAMPLE RECEPTACLE 有权
    样品接收器

    公开(公告)号：US20140056784A1

    公开(公告)日：2014-02-27

    申请号：US14003755

    申请日：2012-03-09

    Applicant: Daniel Simons ,  Dirk Leber ,  Harald Quintel ,  Sasa Lazevski ,  Bruno Walder ,  Andreas Bretscher ,  Thomas Voit

    Inventor： Daniel Simons ,  Dirk Leber ,  Harald Quintel ,  Sasa Lazevski ,  Bruno Walder ,  Andreas Bretscher ,  Thomas Voit

    IPC: B01L3/00

    CPC classification number: B01L3/50825 ,  B01L2300/045 ,  B65D39/06

    Abstract: The invention relates to a sample container, comprising a housing which forms a sample space for receiving a sample and has at least one circular opening which extends in a channel-shaped manner into the sample space, and further comprising a spherical closing element, wherein the diameter of the closing element only exceeds the diameter of the opening channel in at least one (closing) portion to such an extent that the closing element can be fixed in a force-locked manner by its largest circumference in the closing portion, wherein the spherical closing element is in contact with the housing, and the opening channel between the closing portion and the inner opening forms a protrusion which reduces the opening cross section of the opening channel with respect to the opening cross section in the closing portion.

    Abstract translation: 本发明涉及一种样品容器，其包括壳体，其形成用于接收样品的样品空间，并且具有至少一个以通道形状延伸到样品空间中的圆形开口，并且还包括球形闭合元件，其中， 关闭元件的直径仅在至少一个（关闭）部分中超过开口通道的直径，使得闭合元件可以通过其在关闭部分中的最大圆周以锁定方式被固定，其中，球形 关闭元件与壳体接触，并且封闭部分和内部开口之间的开口通道形成一个突起，该突起减小了开口通道相对于封闭部分中的开口横截面的开口横截面。

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