Strategies for silencing human disease using RNA interference.
作者： Daniel H. Kim , John J. Rossi
DOI: 10.1038/NRG2006
关键词: Virus diseases 、 Genetic therapy 、 Treatment options 、 Computational biology 、 Extramural 、 Biology 、 Effector 、 Gene silencing 、 Human disease 、 RNA interference 、 Genetics
摘要: Since the first description of RNA interference (RNAi) in animals less than a decade ago, there has been rapid progress towards its use as therapeutic modality against human diseases. Advances our understanding mechanisms RNAi and studies vivo indicate that RNAi-based therapies might soon provide powerful new arsenal pathogens diseases for which treatment options are currently limited. Recent findings have highlighted both promise challenges using applications. Design delivery strategies effector molecules must be carefully considered to address safety concerns ensure effective, successful
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