Volume 88 Issue 48 | p. 36
Issue Date: November 29, 2010

Mismatch Route To Targeted Therapy

RNA with less than perfect complementarity blocks expression of Huntington’s Disease gene sequence
Department: Science & Technology
Keywords: Huntington's disease, huntingtin protein, siRNA

A new therapeutic strategy for treating Huntington’s disease patients that is based on small interfering RNA (siRNA) could have fewer side effects than earlier approaches (Chem. Biol., DOI: 10.1016/j.chembiol.2010.10.013). The technique uses designed synthetic RNAs that behave counterintuitively: They work better when modified to interact less efficiently with their intended targets.

If it can be replicated in vivo, the work could lead to therapies for Huntington’s disease and similar conditions that result from genetic mutations . . .

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