Becca A. Flitter

1 month ago | science.org | Becca A. Flitter |Huilan Wang |Stephen Brown |Mandeep Singh |Wan-Jin Chen |Beltran Borges | +13 more

Editor’s summaryTreatment of spinal muscular atrophy (SMA) ideally starts in the early postnatal period, but patients with severe forms of SMA may still develop substantial neurological deficits despite this. Here, Borges and colleagues showed that prenatal treatment with antisense oligonucleotides (ASOs) via intra-amniotic injection improved outcomes in two mouse models of severe SMA.