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  • Therapeutic application of RNAi: is mRNA targeting finally ready for prime time?
  • Grimm D; Kay MA
  • J Clin Invest 2007[Dec]; 117 (12): 3633-41
  • With unprecedented speed, RNA interference (RNAi) has advanced from its basic discovery in lower organisms to becoming a powerful genetic tool and perhaps our single most promising biotherapeutic for a wide array of diseases. Numerous studies document RNAi efficacy in laboratory animals, and the first clinical trials are underway and thus far suggest that RNAi is safe to use in humans. Yet substantial hurdles have also surfaced and must be surmounted before therapeutic RNAi applications can become a standard therapy. Here we review the most critical roadblocks and concerns for clinical RNAi transition, delivery, and safety. We highlight emerging solutions and concurrently discuss novel therapeutic RNAi-based concepts. The current rapid advances create realistic optimism that the establishment of RNAi as a new and potent clinical modality in humans is near.
  • |*Drug Delivery Systems[MESH]
  • |Animals[MESH]
  • |Clinical Trials as Topic[MESH]
  • |Drug-Related Side Effects and Adverse Reactions[MESH]
  • |Humans[MESH]
  • |RNA Interference/*drug effects[MESH]
  • |RNA, Messenger/*antagonists & inhibitors[MESH]
  • |RNA, Small Interfering/adverse effects/chemistry/*therapeutic use[MESH]

  • *{{pmid18060021}}
    *<b>[ Therapeutic application of RNAi: is mRNA targeting finally ready for prime time?]</b> J Clin Invest 2007; 117(12) ; 3633-41 Grimm D; Kay MA


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    J Clin Invest

    3633 12.117 2007