Evaluation of the orally bioavailable 4-phenylbutyrate-tethered trichostatin A analogue AR42 in models of spinal muscular atrophy

Proximal spinal muscular atrophy (SMA) is a leading genetic cause for infant death in the world and results from the selective loss of motor neurons in the spinal cord. SMA is a consequence of low levels of SMN protein and small molecules that can increase SMN expression are of considerable interest...

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Detalles Bibliográficos
Autores principales: Lumpkin, Casey J., Harris, Ashlee W., Connell, Andrew J., Kirk, Ryan W., Whiting, Joshua A., Saieva, Luciano, Pellizzoni, Livio, Burghes, Arthur H. M., Butchbach, Matthew E. R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10293174/
https://www.ncbi.nlm.nih.gov/pubmed/37365234
http://dx.doi.org/10.1038/s41598-023-37496-0

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10293174/
https://www.ncbi.nlm.nih.gov/pubmed/37365234
http://dx.doi.org/10.1038/s41598-023-37496-0

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