Systems Biology Investigation of cAMP Modulation to Increase SMN Levels for the Treatment of Spinal Muscular Atrophy

Spinal muscular atrophy (SMA), a leading genetic cause of infant death worldwide, is an autosomal recessive disorder caused by the loss of SMN1 (survival motor neuron 1), which encodes the protein SMN. The loss of SMN1 causes a deficiency in SMN protein levels leading to motor neuron cell death in t...

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Detalles Bibliográficos
Autores principales: Mack, Sean G., Cook, Daniel J., Dhurjati, Prasad, Butchbach, Matthew E. R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2014
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4267815/
https://www.ncbi.nlm.nih.gov/pubmed/25514431
http://dx.doi.org/10.1371/journal.pone.0115473

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4267815/
https://www.ncbi.nlm.nih.gov/pubmed/25514431
http://dx.doi.org/10.1371/journal.pone.0115473

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