Cargando…
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,...
Autores principales: | Meijboom, Katharina E., Volpato, Viola, Monzón-Sandoval, Jimena, Hoolachan, Joseph M., Hammond, Suzan M., Abendroth, Frank, de Jong, Olivier G., Hazell, Gareth, Ahlskog, Nina, Wood, Matthew J.A., Webber, Caleb, Bowerman, Melissa |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society for Clinical Investigation
2021
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8410072/ https://www.ncbi.nlm.nih.gov/pubmed/34236053 http://dx.doi.org/10.1172/jci.insight.149446 |
Ejemplares similares
-
Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy
por: Shabanpoor, Fazel, et al.
Publicado: (2017) -
Light modulation ameliorates expression of circadian genes and disease progression in spinal muscular atrophy mice
por: Walter, Lisa M, et al.
Publicado: (2018) -
Interventions Targeting Glucocorticoid-Krüppel-like Factor 15-Branched-Chain Amino Acid Signaling Improve Disease Phenotypes in Spinal Muscular Atrophy Mice
por: Walter, Lisa M., et al.
Publicado: (2018) -
Dysregulation of Tweak and Fn14 in skeletal muscle of spinal muscular atrophy mice
por: Meijboom, Katharina E., et al.
Publicado: (2022) -
Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy
por: Hammond, Suzan M., et al.
Publicado: (2022)