An acyclic nucleic acid-modified siRNA targeting CAG expansions for polyglutamine disease treatment

Polyglutamine (polyQ) diseases are inherited neurological disorders caused by an expansion of the cytosine-adenine-guanine (CAG) repeat in the causative genes. These include Huntington’s disease, spinal and bulbar muscular atrophy (SBMA), and spinocerebellar ataxias (SCAs). Clinical trials have been...

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Autori principali:	Kentaro Maeda, Tomoki Hirunagi, Kentaro Sahashi, Yukiko Kamiya, Madoka Iida, Kenji Sakakibara, Kazunari Onodera, Manabu Ohyama, Yohei Okada, Hideyuki Okano, Hiroyuki Asanuma, Masahisa Katsuno
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Elsevier 2026-03-01
Serie:	Molecular Therapy: Nucleic Acids
Soggetti:	MT: Oligonucleotides: Therapies and Applications siRNA CAG repeat allele selective polyglutamine diseases SBMA
Accesso online:	http://www.sciencedirect.com/science/article/pii/S2162253125003567
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