Multi-disciplinary approach to validating or indentifying targets using an in vivo system
    4.
    发明申请
    Multi-disciplinary approach to validating or indentifying targets using an in vivo system 审中-公开
    使用体内系统验证或识别目标的多学科方法

    公开(公告)号:US20070237718A1

    公开(公告)日:2007-10-11

    申请号:US11717603

    申请日:2007-03-12

    CPC classification number: G01N33/6803 C12Q1/6886 G01N33/5088 G01N33/574

    Abstract: The present invention embodies a multi-disciplinary approach to validate or identify targets involved in any given biological process or pathway, such as an immune response, or progression or regression of disease. By introducing a target(s) with, e.g., gene delivery vector(s) or other foreign substance(s) to an in vivo system and by integrating, for example, pathological, pharmacological, bioassay, microarray and bioinformatics data obtained from the in vivo system, the present inventors are able to (1) identify one or more targets, e.g., genes, that are involved in a biological pathway of interest, (2) implement these identified targets for further analysis of the biological process or pathway and (3) provide a scalable approach in vivo for potential large quantities of target(s) discovery and validation. This process can be used in any number of applications, including the identification of agonists and antagonists to a biological process or pathway, which can lead to drugs and vaccine discovery.

    Abstract translation: 本发明体现了一种多学科方法来验证或鉴定涉及任何给定生物过程或途径(例如免疫应答或疾病进展或消退)的靶标。 通过将诸如基因递送载体或其他外来物质的靶引入体内系统,并通过整合例如病理学,药理学,生物测定,微阵列和生物信息学数据,从 本发明人能够(1)识别涉及生物学途径的一个或多个目标,例如基因,(2)实施这些鉴定的目标以进一步分析生物过程或途径和( 3)为潜在的大量目标发现和验证在体内提供可扩展的方法。 该方法可用于任何数量的应用,包括鉴定可导致药物和疫苗发现的生物过程或途径的激动剂和拮抗剂。

    Therapeutic methods for nucleic acid delivery vehicles
    5.
    发明申请
    Therapeutic methods for nucleic acid delivery vehicles 审中-公开
    核酸递送载体的治疗方法

    公开(公告)号:US20070219118A1

    公开(公告)日:2007-09-20

    申请号:US11591611

    申请日:2006-11-02

    CPC classification number: A61K48/0041 A61K48/0008 A61K48/0083 A61K48/0091

    Abstract: It has been found that certain synthetic vectors and nucleic acid sequences that encode viral genomic sequences can, for example, be administered to a subject repeatedly as a vehicle for effectively delivering one or more therapeutic nucleic acid molecules or polypeptides to a cell or tissue. Accordingly, the disclosed nucleic acid delivery vehicles can be used, for instance, as part of a therapeutic regimen that involves an ongoing use of a therapeutic nucleic acid molecule or polypeptide.

    Abstract translation: 已经发现,编码病毒基因组序列的某些合成载体和核酸序列可以例如作为用于有效地将一种或多种治疗性核酸分子或多肽有效递送至细胞或组织的载体重复施用于受试者。 因此,所公开的核酸递送载体可用于例如涉及正在使用治疗性核酸分子或多肽的治疗方案的一部分。

    Method of achieving persistent Transgene expression
    7.
    发明申请
    Method of achieving persistent Transgene expression 审中-公开
    实现持续转基因表达的方法

    公开(公告)号:US20090069257A1

    公开(公告)日:2009-03-12

    申请号:US11732158

    申请日:2007-04-02

    CPC classification number: C12N15/87 A61K48/0075 A61K48/0083

    Abstract: Non-inflammatory vector compositions are provided that are suitable for repeated transgene delivery and that result in persistent transgene expression. The compositions are non-inflammatory, the present compositions are suitable for readministration and do not induce expression-limiting immune or inflammatory responses. Thus, these compositions are useful in methods of repeated administration to achieve persistent transgene expression, and are especially suited to treating genetic, acquired and inflammation-associated conditions.

    Abstract translation: 提供非炎症载体组合物,其适用于重复的转基因递送,并导致持续的转基因表达。 组合物是非炎性的,本发明的组合物适用于再次给药并且不诱导表达限制性免疫或炎症反应。 因此,这些组合物可用于重复施用以实现持续转基因表达的方法,并且特别适合于治疗遗传,获得性和炎症相关病症。

    Compositions for treating respiratory viral infections and their use
    9.
    发明授权
    Compositions for treating respiratory viral infections and their use 有权
    用于治疗呼吸道病毒感染及其用途的组合物

    公开(公告)号:US08691781B2

    公开(公告)日:2014-04-08

    申请号:US11792179

    申请日:2005-11-04

    Abstract: The invention provides siRNA compositions that interfere with viral replication in respiratory viral infections, including respiratory syncytial virus and avian influenza A, including the H5N1 strain. The invention further provides uses of the siRNA compositions to inhibit expression of viral genes in respiratory virus-infected cells, and to uses in the treatment of respiratory virus infections in a subject. Generally the invention provides polynucleotide that includes a first nucleotide sequence of 15 to 30 bases that targets the genome of a respiratory syncytial virus or an influenza A virus, a complement thereof, a double stranded polynucleotide or a hairpin polynucleotide. Additionally the invention provides vectors, cells and pharmaceutical compositions containing siRNA sequences.

    Abstract translation: 本发明提供干扰呼吸道病毒感染(包括呼吸道合胞病毒和禽流感A)包括H5N1毒株在内的病毒复制的siRNA组合物。 本发明还提供siRNA组合物用于抑制呼吸道病毒感染细胞中病毒基因表达的用途,以及用于治疗受试者中的呼吸道病毒感染的用途。 本发明通常提供多核苷酸,其包含15至30个碱基的第一核苷酸序列,其靶向呼吸道合胞病毒或甲型流感病毒的基因组,其补体,双链多核苷酸或发夹多核苷酸。 另外,本发明提供了含有siRNA序列的载体,细胞和药物组合物。

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