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Innovating spinal muscular atrophy models in the therapeutic era

Spinal muscular atrophy (SMA) is a severe, monogenetic, neuromuscular disease. A thorough understanding of its genetic cause and the availability of robust models has led to the development and approval of three gene-targeting therapies. This is a unique and exciting development for the field of neu...

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Detalles Bibliográficos
Autores principales: Signoria, Ilaria, van der Pol, W. Ludo, Groen, Ewout J. N.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Company of Biologists Ltd 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10565113/
https://www.ncbi.nlm.nih.gov/pubmed/37787662
http://dx.doi.org/10.1242/dmm.050352