99. Antisense oligonucleotide jacifusen for FUS-ALS: an investigator-initiated, multicentre, open-label case series.
作者: Neil A Shneider.;Matthew B Harms.;Vlad A Korobeynikov.;Olivia M Rifai.;Benjamin N Hoover.;Elizabeth A Harrington.;Sonya Aziz-Zaman.;Jessica Singleton.;Arish Jamil.;Vikram R Madan.;Ikjae Lee.;Jinsy A Andrews.;Richard M Smiley.;Mahabub M Alam.;Lauren E Black.;Minwook Shin.;Jonathan K Watts.;David Walk.;Daniel Newman.;Robert M Pascuzzi.;Markus Weber.;Christoph Neuwirth.;Sandrine Da Cruz.;Armand Soriano.;Roger Lane.;Scott Henry.;Joel Mathews.;Paymaan Jafar-Nejad.;Dan Norris.;Frank Rigo.;Robert H Brown.;Stephan Miller.;Rebecca Crean.;C Frank Bennett.
来源: Lancet. 2025年405卷10494期2075-2086页
Pathogenic variants of fused in sarcoma (FUS) cause amyotrophic lateral sclerosis (FUS-ALS), with evidence of gain of function. Jacifusen is an antisense oligonucleotide targeting FUS pre-mRNA, previously shown to delay neurodegeneration in a mouse model and potentially slow functional decline in a first-in-human study. Here, we sought to further evaluate use of jacifusen as a treatment for FUS-ALS.
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