Dysregulation of ubiquitin homeostasis and β-catenin signaling promote spinal muscular atrophy

Thomas M. Wishart, Chantal A. Mutsaers, Michell M. Reimer, +20

Journal of Clinical Investigation

Molecular RegulationSynaptic SignalingCellular PhysiologyTranscriptional RegulationNeuroregeneration +20

Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy

Rachael A. Powis, Evangelia Karyka, Penelope J Boyd, +13

JCI Insight

SMN-dependent intrinsic defects in Schwann cells in mouse models of spinal muscular atrophy

Gillian Hunter, Arwin Aghamaleky Sarvestany, Sarah L. Roche, +2

Human Molecular Genetics

Schwann CellsKey Myelin ProteinsMyelination DefectsPeripheral NervePeripheral Nerves +23

Label-Free Quantitative Proteomic Profiling Identifies Disruption of Ubiquitin Homeostasis As a Key Driver of Schwann Cell Defects in Spinal Muscular Atrophy

Arwin Aghamaleky Sarvestany, Gillian Hunter, Amy Tavendale, +5

Journal of Proteome Research

GeneticsCytoskeletonLow LevelsUbiquitin PathwaysProteomic Technology +15

Restoration of SMN in Schwann cells reverses myelination defects and improves neuromuscular function in spinal muscular atrophy

Gillian Hunter, Rachael A. Powis, Ross A. Jones, +7

Human Molecular Genetics

Label-free proteomics identifies Calreticulin and GRP75/Mortalin as peripherally accessible protein biomarkers for spinal muscular atrophy

Chantal A. Mutsaers, Douglas J. Lamont, Gillian Hunter, +2

Genome Medicine

Increased levels of <scp>UCHL</scp>1 are a compensatory response to disrupted ubiquitin homeostasis in spinal muscular atrophy and do not represent a viable therapeutic target

Rachael A. Powis, Chantal A. Mutsaers, Thomas M. Wishart, +3

Neuropathology and Applied Neurobiology

Clinical response and on-treatment clinical remission with tezepelumab in a broad population of patients with severe, uncontrolled asthma: results over 2 years from the NAVIGATOR and DESTINATION studies

Michael E. Wechsler, Guy Brusselle, J. Christian Virchow, +12

European Respiratory Journal

Microvasculopathy in spinal muscular atrophy is driven by a reversible autonomous endothelial cell defect

Haiyan Zhou, Ying Hong, Mariacristina Scoto, +19

Journal of Clinical Investigation

Vascular DiseasePathologyBiomedical EngineeringRegenerative MedicineAngiogenesis +16

Evaluation of Antibody Properties and Clinically Relevant Immunogenicity, Anaphylaxis, and Hypersensitivity Reactions in Two Phase III Trials of Tralokinumab in Severe, Uncontrolled Asthma

Mats Carlsson, Martin Braddock, Yuling Li, +6

Drug Safety