• 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

        Pharmacogenomics of Rare and Monogenic Disorders

        互联网

        325
        The rare monogenic diseases provide the most clearly evident examples of pathology resulting from a single genetic lesion. As such, they are in some sense the “low hanging fruit” for the application of pharmacogenomic therapeutic approaches. These quite often serious diseases, while still not fully understood, have seen a revolution in both disease classification as well as therapeutic approaches. Advances in genomic understanding of rare diseases both challenge traditional disease classifications as well as reveal, in many instances, a complex interplay of the host genome with its environment. Therapeutic approaches initially developed as “orphan” products, including molecular chaperones, agents to promote or stop codon skipping, various gene therapy techniques, substrate reduction therapies, and other novel therapies, have all either recently seen market approval or show clear promise as potential future treatment approaches.
        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