Friday, December 11, 2015

Stepping Toward Therapeutic use of CRISPR

Stepping toward therapeutic CRISPR


Gagnon et al

Most new technologies for manipulating gene expression in mammalian cells are accepted at a relatively slow pace. Occasionally, however, a new technology is so robust and fills such a critical niche that its adoption is widespread and rapid. Fifteen years ago, duplex RNAs were such a technology. RNA interference (RNAi) in mammalian cells was first demonstrated in 2001 (1) and within 2 y RNAi was a commonly used tool throughout industry and academia. RNAi is making its way into clinical trials as a potential therapeutic as challenges in delivery to relevant tissues begin to be overcome (2–4).

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