This week, Díaz-Hernández et al. try to arrest Huntington's disease (HD) late in the course by silencing mutant huntingtin (htt) in a mouse model of HD (Tet/HD94).
Despite progressive loss of motor coordination, striatal atrophy, and neuronal htt-positive inclusions, the Tet/HD94 mice do not show loss of striatal neurons until 17 months of age.
At this late stage, the authors treated the conditional mutant mice with doxycycline to shut off expression of the htt transgene. After five additional months, "gene-off" mice showed improved motor performance, whereas untreated "gene-on" Tet/HD94 mice continued to worsen.
The authors suggest that gene silencing even at a late stage could have therapeutic potential.
Miguel Díaz-Hernández, Jesús Torres- Peraza, Alejandro Salvatori-Abarca, María A. Morán, Pilar Gómez-Ramos, Jordi Alberch, and José J. Lucas
Sara Harris
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