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Exon 1-Targeting Mirna Reduces the Pathogenic Exon 1 HTT Protein in Huntington's Disease Models

Marina Sogorb-Gonzalez,Christian Landles,Nicholas S. Caron,Anouk Stam,Georgina Osborne,Michael R. Hayden,David Howland,Sander van Deventer,Gillian P. Bates,Astrid Valles,Melvin Evers

BRAIN（2024）

引用 0|浏览3
关键词
Huntington's disease,aberrant splicing,HTT exon 1,AAV5-miHTT,gene therapy,Huntington's disease mouse models
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