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

Muiños-Bühl, A; Rombo, R; Ling, KK; Zilio, E; Rigo, F; Bennett, CF; Wirth, B

Wirth, B (通讯作者)，Univ Cologne, Inst Human Genet, D-50931 Cologne, Germany.;Wirth, B (通讯作者)，Univ Cologne, Ctr Mol Med Cologne, D-50931 Cologne, Germany.;Wirth, B (通讯作者)，Univ Hosp Cologne, Ctr Rare Dis Cologne, D-50931 Cologne, Germany.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2023; 24 (4):

Abstract

For SMA patients with only two SMN2 copies, available therapies might be insufficient to counteract lifelong motor neuron (MN) dysfunction. Therefore,......

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