Long-Term Safety and Efficacy of AAV9 Vectors Expressing Human SMN1 Gene: A Preclinical Study
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Spinal muscular atrophy (SMA) is a severe genetic neuromuscular disorder caused by deletions or mutations in the SMN1 gene, leading to reduced levels of the survival motor neuron (SMN) protein. Gene therapy using adeno-associated virus serotype 9 (AAV9) has em... ...
