Spinal muscular atrophy (SMA) is a common inherited disease of childhood characterized by progressive degeneration of motor neurons (MN) and muscle atrophy. In most of the cases, SMA is due to homozygous mutations in the Survival of Motor Neuron gene (SMN1), leading to reduced levels of the ubiquitous protein SMN. To date, SMN has been mainly involved in RNA metabolism especially in MN. We and others have demonstrated that a single intravenous injection of SMN1-encoding scAAV9 vectors (scAAV9-SMN1) induced a tremendous rescue of SMND7 mice, a common model of SMA.

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