• 我要登录|
  • 免费注册
    |
  • 我的丁香通
    • 企业机构:
    • 成为企业机构
    • 个人用户:
    • 个人中心
  • 移动端
    移动端
丁香通 logo丁香实验_LOGO
搜实验

    大家都在搜

      大家都在搜

        0 人通过求购买到了急需的产品
        免费发布求购
        发布求购
        点赞
        收藏
        wx-share
        分享

        Clinical Aspects of Ribozymes as Therapeutics in Gene Therapy

        互联网

        1033
        By June 1995, 112 gene therapy trials had been approved by the NIH Recombinant DNA Advisory Committee (RAC) in the US, involving 500 actual, planned, or projected patients ( 13 ). Marking trials comprise roughly 20% (25 in total) of all approved studies, while the remainder are considered to have therapeutic objectives. All together 87 treatment protocols are active, including 9 trials directed toward treatment of HIV infection, 21 that address genetic disorders, 1 each involving treatment of autoimmune or atherosclerotic disease, and 55 studies that target cancer therapy in some form ( see Fig. 1 ). Therapeutic trials include 38 in vivo studies and 42 ex vivo studies, all together employing over 81 vectors (50 murine, 15 adenovirus, 1 AAV), as well as several other delivery systems (12 lipid mediated and 3 naked DNA delivery protocols). Treatment studies involving protein expression include replacement studies (e.g., ADA deficiency, cystic fibrosis, α-1-antitrypsin deficiency, Gaucher’s disease, familial hypercholesterolemia, and Hunter syndrome), and trials aimed to produce immunomodulation or immunoprophylaxis (including introduction of genetically engineered cytotoxic lymphocytes, vectors expressing viral proteins, and vectors expressing cytokines, such as GM-CSF, IL-2, IL-3, IL-6, IL-7, IL-12, and TNF, into malignant cells [ 13 ). Other trials involved introduction of toxin genes, particularly thymidine kinase ( hsv-tk ), into tumor tissue or protective genes (e.g., human multidrug resistance gene) into normal hematopoetic tissue to protect bone marrow during cytotoxic chemotherapy.
         
        Fig 1.  Approved gene therapy trials in the US Data from the Office of Recombinant DNA Activities (ORDA) as published in Human Gene Therapy summaries in Gene Therapy Newsletter Issue 12, pp 1–2 (1995) was used to construct a cumulative graph indicating active approved protocols in the indicated areas. The group designated as “Other” includes protocols for autoimmune diseases and atherosclerotic vascular disease, and may not include protocols for which RAC approval was deferred with subsequent submission directly to the FDA.

        ad image
        提问
        扫一扫
        丁香实验小程序二维码
        实验小助手
        丁香实验公众号二维码
        扫码领资料
        反馈
        TOP
        打开小程序