换一批Mitochondria replacement from transplanted amniotic fluid stem cells:a promising therapy for non-neuronal defects in spinal muscular atrophy
摘要Spinal muscular atrophy (SMA) is a genetic disorder that primarily affects infants and leads to muscle weakness, atrophy, and paralysis. The main cause is the homozygous mutation or deletion of the SMN1 gene, resulting in inadequate levels of the survival motor neuron (SMN) protein. Approved treatments focus on restoring SMN levels through various approaches, but there is a need for "SMN-independent" therapies that target other pathological processes.
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