In SMA murine models, the administration of a «self-complementary» adenovirus serotype 9-associated virus (scAAV9) vector containing one or more copie(s) of the SMN transgene (scAAV9-SMN) showed its ability to cross the blood brain barrier and achieve high levels of neuronal transduction39–41. The gene discussed is SMN1; the disease is proximal spinal muscular atrophy.