Vectors for tissue-specific replication
    7.
    发明申请
    Vectors for tissue-specific replication 审中-公开
    用于组织特异性复制的载体

    公开(公告)号:US20080213884A1

    公开(公告)日:2008-09-04

    申请号:US11977902

    申请日:2007-10-25

    IPC分类号: C12N5/06

    摘要: The invention generally relates to targeted gene therapy using recombinant vectors and particularly adenovirus vectors. The invention specifically relates to replication-conditional vectors and methods for using them. Such vectors are able to selectively replicate in a target tissue to provide a therapeutic benefit from the presence of the vector per se or from heterologous gene products expressed from the vector and distributed throughout the tissue. In such vectors, a gene essential for replication is placed under the control of a heterologous tissue-specific transcriptional regulatory sequence. Thus, replication is conditioned on the presence of a factor(s) that induces transcription or the absence of a factor(s) that inhibits transcription of the gene by means of the transcriptional regulatory sequence with this vector, therefore, a target tissue can be selectively treated.

    摘要翻译: 本发明一般涉及使用重组载体,特别是腺病毒载体的靶向基因治疗。 本发明具体涉及复制条件向量及其使用方法。 这样的载体能够在靶组织中选择性地复制以从载体本身的存在或从载体表达的分布在整个组织中的异源基因产物提供治疗益处。 在这样的载体中,将复制必需的基因置于异源组织特异性转录调控序列的控制之下。 因此,复制受到诱导转录的因子的存在或不存在通过该载体通过转录调节序列抑制基因转录的因子的因素,因此,靶组织可以是 选择性处理。

    Compositions and methods for the enhanced uptake of therapeutic agents through the bladder epithelium
    8.
    发明申请
    Compositions and methods for the enhanced uptake of therapeutic agents through the bladder epithelium 审中-公开
    通过膀胱上皮增强治疗剂吸收的组合物和方法

    公开(公告)号:US20080076703A1

    公开(公告)日:2008-03-27

    申请号:US11891659

    申请日:2007-08-09

    摘要: Compositions and methods for enhancing introduction of therapeutic agents into the bladder epithelium for the treatment of bladder diseases and disorders such as bladder cancer are described. According to one method, the luminal surface of the bladder is contacted with a composition comprising a bladder enhancer and a therapeutic agent for the treatment of the bladder disease. According to an alternative method, the luminal surface of the bladder is first contacted with a pretreatment composition comprising a bladder enhancer and subsequently contacted with a composition comprising a therapeutic agent for the treatment of the bladder disease. The transduction enhancing agent can be a mono-, di-, or poly-saccharide having a lipophilic substituent such as n-dodecyl-β-D-maltoside (DDM). Compositions comprising a transduction enhancing agent and a therapeutic agent for the treatment of a bladder disease are also described.

    摘要翻译: 描述了用于增强将治疗剂引入膀胱上皮以治疗膀胱疾病和疾病如膀胱癌的组合物和方法。 根据一种方法,膀胱的管腔表面与包含膀胱增强剂和治疗膀胱疾病的治疗剂的组合物接触。 根据另一种方法,首先将膀胱的腔表面与包含膀胱增强剂的预处理组合物接触,随后与包含用于治疗膀胱疾病的治疗剂的组合物接触。 转导增强剂可以是具有亲脂性取代基如正十二烷基-β-D-麦芽糖苷(DDM)的单糖,二糖或多糖。 还描述了包含转导增强剂和用于治疗膀胱疾病的治疗剂的组合物。