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Targeted knockdown of Smn in muscle stem cells induces non-cell autonomous loss of motor neurons

Jordan Mecca et al.
HAL (France) · Papers
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Abstract Spinal muscular atrophy (SMA) is due to a deficit in SMN, a ubiquitously expressed protein encoded by the Survival of Motor Neuron 1 (SMN1) gene. Recently, SMN-targeted disease-modifying treatments have greatly improved the clinical outcomes of this neuromuscular disease. However, uncertain
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