Molecular Therapy - Nucleic Acids
Volume 24, 4 June 2021, Pages 1-10
Original ArticleSelective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS
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open access
Graphical abstract
Keywords
siRNA
CAG repeat
selective suppression
polyglutamine diseases
SBMA
androgen receptor
unlocked nucleic acid
lipid nanoparticle
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© 2021 The Author(s).