Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS
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H. Okano | R. Mukthavaram | Y. Okada | M. Ohyama | P. Karmali | M. Katsuno | K. Sahashi | M. Iida | G. Tohnai | Kazunari Onodera | K. Tachikawa | Michelle Nguyen | P. Chivukula | Tomoki Hirunagi | Angel Leu | Rajesh Mukthavaram