Lowering of Toxic Dipeptide Proteins and Phenotype Rescue in an ALS Mouse Model Treated with AAV-miQURE? Targeting the Repeat Expansion-Containing C9orf72 Transcripts

Vanessa Zancanella, Anna-Aster de Ruiter, Romy M. van Vliet,Kristel S. van Rooijen,Madhurima Chatterjee,Ines Lopes Pereira, Vlad Shtefaniuk, Sebastian N. Kieper, Irena Bockaj,Morgane Wartel,Elena de Miguel,Greg Dobrynin, Mercedes Valls Seron, Jovana Kovacevic,Ying Poi Liu

MOLECULAR THERAPY(2023)

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