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Targeting the Sense and Antisense Pathogenic C9ORF72 Transcripts As Therapy for C9ORF72 ALS

Vanessa Zancanella,Kristel van Rooijen,Ines Lopes Pereira,Cynthia Brouwers, Noortje Heidinga, Soraya Smit, Warner Hoornenborg, Rhode Erbrink,Irena Bockaj,Greg Dobrynin, Jovana Kovacevic,Morgane Wartel,Stijn Waaijer, Tycho Hoogland, Mercedes Valls Seron,Melvin M. Evers,Ying Poi Liu

MOLECULAR THERAPY（2024）

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