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Ramee Lee

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Blocking somatic repeat expansion and lowering huntingtin by RNAi synergize to attenuate Huntington’s disease pathogenesis in mice

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By Kimberly A. Dowd, Huaxin Song, Saif Malik, Jillian Belgrad, Ashley Summers, Christian Landles, Jonathan Greene, Samuel R. Hildebrand, Emily Knox, Ellen Sapp, Raymond C. Furgal, Rachael Miller, Georgina Osborne, Kathryn Chase, Eric Luu, Jason Freedman, Brianna Bramato, Nicholas McHugh, Daniel O’Reilly, Paul L. Greer, Gillian Bates, Thomas Vogt, Nozomi Yamada, Vicky Benoit, Ramee Lee
| Science Magazine Verified
Editor’s summary Abstract Supplementary Materials REFERENCES AND NOTES Information & Authors Metrics & Citations Check Access References Figures Tables Media Share Editor’s summary The DNA repair protein MSH3 drives somatic CAG repeat expansion in the huntingtin gene (HTT), and both MSH3 and HTT are key of RNA therapeutics for Huntington’s disease (HD). In a series of experiments in a mouse model of HD, Belgrad et al.
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