Long-Term SMN- and Ncald-ASO Combinatorial Therapy in SMA Mice and NCALD-ASO Treatment in hiPSC-Derived Motor Neurons Show Protective Effects

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Authors: Anixa Muiños-Bühl, Roman Rombo, Karen K. Ling, Eleonora Zilio, Frank Rigo, C. Frank Bennett, Brunhilde Wirth

Journal title: International Journal of Molecular Sciences

Journal number: 24

Journal publisher: Multidisciplinary Digital Publishing Institute (MDPI)

Published year: 2023

Published pages: 4198

DOI identifier: 10.3390/ijms24044198

ISSN: 1422-0067