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

For SMA patients with only two SMN2 copies, available therapies might be insufficient to counteract lifelong motor neuron (MN) dysfunction. Therefore, additional SMN-independent compounds, supporting SMN-dependent therapies, might be beneficial. Neurocalcin delta (NCALD) reduction, an SMA protective...

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Detalles Bibliográficos
Autores principales: Muiños-Bühl, Anixa, Rombo, Roman, Ling, Karen K., Zilio, Eleonora, Rigo, Frank, Bennett, C. Frank, Wirth, Brunhilde
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9961752/
https://www.ncbi.nlm.nih.gov/pubmed/36835624
http://dx.doi.org/10.3390/ijms24044198

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9961752/
https://www.ncbi.nlm.nih.gov/pubmed/36835624
http://dx.doi.org/10.3390/ijms24044198

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