Title: | Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS |
Journal : | Molecular therapy. Nucleic acids, 24 |
Authors: | Hirunagi T ; Sahashi K ; Tachikawa K ; Leu AI ; Nguyen M ; Mukthavaram R ; Karmali PP ; Chivukula P ; Tohnai G ; Iida M ; Onodera K ; Ohyama M ; Okada Y ; Okano H ; Katsuno M |
Material Type: | Article |
Publication Date: | 15/02/2021 |
Size: | p 1 |
Languages: | English |
Keywords: | CAG repeat;SBMA;androgen receptor;lipid nanoparticle;polyglutamine diseases;selective suppression;siRNA;unlocked nucleic acid; |
Pubmed / DOI : | Pubmed : 33738134 / DOI : 10.1016/j.omtn.2021.02.007 |
N° Profil MNM : | 2021032 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/33738134 |