Interventionally targeting somatic CAG expansions can be a rapid disease-modifying therapeutic avenue: Preclinical evidence.
干预性靶向体细胞 CAG 扩增可能是一种快速改变疾病的治疗途径:临床前证据
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doi:10.1101/2025.04.25.650652
Gall-Duncan Terence, Ko Sangyoon Y, Quick Isabelle K, Khan Mahreen, Feng Kristie, Kelley Chase P, Coleman Annabelle, Touze Alexiane, Tang Shuqian, Mehkary Mustafa, Yokoi Katsuyuki, Herrington Casey R, You Justin, Lambie Scott C, Prasolava Tanya K, Panigrahi Gagan B, Park Jeehye, Nakatani Kazuhiko, Byrne Lauren M, Wang Peixiang, Schneekloth John S Jr, Nakamori Masayuki, Frankland Paul W, Wang Eric T, Pearson Christopher E