Antisense oligonucleotide jacifusen for FUS-ALS: an investigator-initiated, multicentre, open-label case series
针对FUS-ALS的反义寡核苷酸jacifusen：一项研究者发起的多中心、开放标签病例系列研究
期刊:Lancet
影响因子:88.5
doi:10.1016/S0140-6736(25)00513-6
Shneider, Neil A; Harms, Matthew B; Korobeynikov, Vlad A; Rifai, Olivia M; Hoover, Benjamin N; Harrington, Elizabeth A; Aziz-Zaman, Sonya; Singleton, Jessica; Jamil, Arish; Madan, Vikram R; Lee, Ikjae; Andrews, Jinsy A; Smiley, Richard M; Alam, Mahabub M; Black, Lauren E; Shin, Minwook; Watts, Jonathan K; Walk, David; Newman, Daniel; Pascuzzi, Robert M; Weber, Markus; Neuwirth, Christoph; Da Cruz, Sandrine; Soriano, Armand; Lane, Roger; Henry, Scott; Mathews, Joel; Jafar-Nejad, Paymaan; Norris, Dan; Rigo, Frank; Brown, Robert H; Miller, Stephan; Crean, Rebecca; Bennett, C Frank
