Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is the leading genetic cause of infant death. We previously developed a high-throughput assay that employs an SMN2-luciferase reporter allowing identification of compounds that act transcriptionally, enhance exon recognition, or stabilize the SMN protein. We describe op...

Fuld beskrivelse

Na minha lista:
Bibliografiske detaljer
Udgivet i:J Med Chem
Main Authors: Rietz, Anne, Li, Hongxia, Quist, Kevin M., Cherry, Jonathan J., Lorson, Christian L., Burnett, Barrington, Kern, Nicholas L., Calder, Alyssa N., Fritsche, Melanie, Lusic, Hrvoje, Boaler, Patrick J., Choi, Sungwoon, Xing, Xuechao, Glicksman, Marcie A., Cuny, Gregory D., Androphy, Elliot J., Hodgetts, Kevin J.
Format: Artigo
Sprog:Inglês
Udgivet: 2017
Fag:
Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5920559/
https://ncbi.nlm.nih.gov/pubmed/28481536
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/acs.jmedchem.6b01885
Tags: Tilføj Tag
Ingen Tags, Vær først til at tagge denne postø!

Lignende værker