Spinal muscular atrophy (SMA) is a neuromuscular disorder characterized by degeneration of the lower motor neurons and progressive muscle weakness leading to paralysis and premature death in the most severe cases. Recently, we developed an efficient gene therapy approach for this disease. In particular, we rescued the severe SMA mouse model, SMNΔ7, using one single intravenous (IV) injection of a self-complementary adeno-associated serotype 9 viral vector (scAAV9) encoding the SMN1 gene under the control of an ubiquitous promoter (PGK).

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