Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy

Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy

Spinal muscular atrophy (SMA) is a neuromuscular disorder caused by loss of survival motor neuron (SMN) protein. While SMN restoration therapies are beneficial, they are not a cure. We aimed to identify potentially novel treatments to alleviate muscle pathology combining transcriptomics, proteomics,...

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Bibliographic Details
Main Authors: Katharina E. Meijboom, Viola Volpato, Jimena Monzón-Sandoval, Joseph M. Hoolachan, Suzan M. Hammond, Frank Abendroth, Olivier G. de Jong, Gareth Hazell, Nina Ahlskog, Matthew J.A. Wood, Caleb Webber, Melissa Bowerman
Format: Article
Language:English
Published: American Society for Clinical investigation 2021-07-01
Series:JCI Insight
Subjects:
Muscle biology
Neuroscience
Online Access:https://doi.org/10.1172/jci.insight.149446

Internet

https://doi.org/10.1172/jci.insight.149446

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