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System-Wide Proteomic Remodeling in Spinal Muscular Atrophy Reveals Tissue-Specific Responses and Partial Rescue by SMN Restoration

2026-03-31

Abstract excerpt

Spinal muscular atrophy (SMA), traditionally defined as a neuromuscular disorder characterized by degeneration of lower motor neurons, is increasingly recognized as a multi-organ disease. SMA is caused by deficiency of the survival motor neuron (SMN) protein below a critical threshold required for cellular homeostasis. While motor neurons are particularly vulnerable, the ubiquitous expression and fundamental funct...

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Literature Corpus work
1e72c41f-60f8-5c12-b19f-5d195882d0e6
DOI
10.64898/2026.03.30.715402
Open publication

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System-Wide Proteomic Remodeling in Spinal Muscular Atrophy Reveals Tissue-Specific Responses and Partial Rescue by SMN RestorationDOI 10.64898/2026.03.30.715402
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