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

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

Polyglutamine (polyQ) diseases are inherited neurodegenerative disorders caused by expansion of cytosine-adenine-guanine (CAG)-trinucleotide repeats in causative genes. These diseases include spinal and bulbar muscular atrophy (SBMA), Huntington’s disease, dentatorubral-pallidoluysian atrophy, and s...

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Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: Elsevier 2021-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
siRNA
CAG repeat
selective suppression
polyglutamine diseases
SBMA
androgen receptor
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253121000421

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http://www.sciencedirect.com/science/article/pii/S2162253121000421

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