Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS

Tomoki Hirunagi, Kentaro Sahashi, Kiyoshi Tachikawa, Angel I. Leu, Michelle Nguyen, Rajesh Mukthavaram, Priya P. Karmali, Padmanabh Chivukula, Genki Tohnai, Madoka Iida, Kazunari Onodera, Manabu Ohyama, Yohei Okada, Hideyuki Okano, Masahisa Katsuno

研究成果: Article査読

1 被引用数 (Scopus)

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Medicine & Life Sciences

Chemical Compounds