Combining multi-omics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy
(2021)
Journal Article
Bowerman. (2021). Combining multi-omics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy. JCI insight, 1-24. https://doi.org/10.1172/jci.insight.149446
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 novel treatments to alleviate muscle pathology... Read More about Combining multi-omics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy.