Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS
| Autor: | Hirunagi, Tomoki, Sahashi, Kentaro, Tachikawa, Kiyoshi, Leu, Angel I., Nguyen, Michelle, Mukthavaram, Rajesh, Karmali, Priya P., Chivukula, Padmanabh, Tohnai, Genki, Iida, Madoka, Onodera, Kazunari, Ohyama, Manabu, Okada, Yohei, Okano, Hideyuki, Katsuno, Masahisa |
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| Zdroj: | In Molecular Therapy - Nucleic Acids 4 June 2021 24:1-10 |
| Databáze: | ScienceDirect |
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