From bench to bedside-the conversion of mirror-image oligonucleotides into therapeutics

来源 :The 2nd Canton Nucleic Acids Forum(第二届广州核酸国际论坛) | 被引量 : 0次 | 上传用户:lcc54202346
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  On the basis of an in vitro evolutionary process, mirror-image RNA or DNA oligonucleotides can be identified to bind and inhibit pharmacologically relevant target molecules conceptually similar to monoclonal antibodies.Such molecules are termed L-aptamers or Spiegelmers.Several Spiegelmer against a variety of targets have been identified.Currently, three Spiegelmer-based compounds are evaluated in clinical studies.So far, Spiegelmers have been shown to be safe and well tolerated.Moreover, strong signs of efficacy in patients have been reported.The identification of a Spiegelmer candidate and its preclinical and early clinical characterization will be presented.
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