Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo

Gene Therapy, Published online: 26 September 2023; doi:10.1038/s41434-023-00418-wArtificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo

https://www.nature.com/articles/s41434-023-00418-w

Source: Gene Therapy - September 26, 2023 Category: Genetics & Stem Cells Authors: Gabriela Toro Cabrera Katharina E. Meijboom Abbas Abdallah Helene Tran Zachariah Foster Alexandra Weiss Nicholas Wightman Rachel Stock Tania Gendron Alisha Gruntman Anthony Giampetruzzi Leonard Petrucelli Robert H. Brown Jr Christian Mueller Source Type: research

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