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Artificial microRNA suppresses C9ORF72variants and decreases toxic dipeptide repeat proteins in vivo

Autor: Cabrera, Gabriela Toro, Meijboom, Katharina E., Abdallah, Abbas, Tran, Helene, Foster, Zachariah, Weiss, Alexandra, Wightman, Nicholas, Stock, Rachel, Gendron, Tania, Gruntman, Alisha, Giampetruzzi, Anthony, Petrucelli, Leonard, Brown, Robert H., Mueller, Christian

Publikováno v: Gene Therapy; 20230101, Issue: Preprints p1-14, 14p

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

Autor: Cabrera GT; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.; Department of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA., Meijboom KE; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA.; Department of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA., Abdallah A; Department of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA., Tran H; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA., Foster Z; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA., Weiss A; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA., Wightman N; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA., Stock R; Department of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA., Gendron T; Department of Neuroscience, Mayo Clinic, 4500 San Pablo Rd., Jacksonville, FL, 32224, USA., Gruntman A; Department of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA., Giampetruzzi A; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA., Petrucelli L; Department of Neuroscience, Mayo Clinic, 4500 San Pablo Rd., Jacksonville, FL, 32224, USA., Brown RH Jr; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA. Robert.Brown@umassmed.edu., Mueller C; Department of Pediatrics and Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA, 01655, USA. Christian.mueller4@sanofi.com.

Publikováno v: Gene therapy [Gene Ther] 2024 Mar; Vol. 31 (3-4), pp. 105-118. Date of Electronic Publication: 2023 Sep 26.