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First proof of principle for treating rare bone disease
Date:11/9/2011

atric patients and normal controls, usually non-affected siblings, in the ongoing "FOP Good Tooth Fairy Program."

The authors caution that the utility of the RNAi approach must be confirmed in mouse models of classic FOP prior to its consideration for human use. Additionally, other hurdles stand in the way of human application at the present time, most notably safe delivery of the small RNA molecules to cells in the human body.

The authors acknowledge that they have a long way to go, but have taken a big first step. "Improvements in RNAi design are advancing at a rapid rate and will enhance the stability, potency, and specificity of inhibitory RNAs, allowing for long-term experiments both in vitro and in vivo," says Shore.


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Contact: Karen Kreeger
karen.kreeger@uphs.upenn.edu
215-349-5658
University of Pennsylvania School of Medicine
Source:Eurekalert  

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