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,...
| Main Authors: | , , , , , , , , , , , |
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| 格式: | Journal article |
| 语言: | English |
| 出版: |
American Society for Clinical Investigation
2021
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