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

• Main Authors: Tomoki Hirunagi (Author), Kentaro Sahashi (Author), Kiyoshi Tachikawa (Author), Angel I. Leu (Author), Michelle Nguyen (Author), Rajesh Mukthavaram (Author), Priya P. Karmali (Author), Padmanabh Chivukula (Author), Genki Tohnai (Author), Madoka Iida (Author), Kazunari Onodera (Author), Manabu Ohyama (Author), Yohei Okada (Author), Hideyuki Okano (Author), Masahisa Katsuno (Author)
• Format: Book
• Published: Elsevier, 2021-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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,...