In vitro and in vivo effects of 2,4 diaminoquinazoline inhibitors of the decapping scavenger enzyme DcpS: Context-specific modulation of SMN transcript levels

C5-substituted 2,4-diaminoquinazoline inhibitors of the decapping scavenger enzyme DcpS (DAQ-DcpSi) have been developed for the treatment of spinal muscular atrophy (SMA), which is caused by genetic deficiency in the Survival Motor Neuron (SMN) protein. These compounds are claimed to act as SMN2 tra...

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Detalles Bibliográficos
Autores principales: Cherry, Jonathan J., DiDonato, Christine J., Androphy, Elliot J., Calo, Alessandro, Potter, Kyle, Custer, Sara K., Du, Sarah, Foley, Timothy L., Gopalsamy, Ariamala, Reedich, Emily J., Gordo, Susana M., Gordon, William, Hosea, Natalie, Jones, Lyn H., Krizay, Daniel K., LaRosa, Gregory, Li, Hongxia, Mathur, Sachin, Menard, Carol A., Patel, Paraj, Ramos-Zayas, Rebeca, Rietz, Anne, Rong, Haojing, Zhang, Baohong, Tones, Michael A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2017
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5612656/
https://www.ncbi.nlm.nih.gov/pubmed/28945765
http://dx.doi.org/10.1371/journal.pone.0185079

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