title: Raise the Roof: Boosting the Efficacy of a Spinal Muscular Atrophy Therapy.
abstract: Spinal muscular atrophy is the most common genetic killer of infants. A therapy shows promise in the clinic, but there is a potential limit to its efficacy. In this issue of Neuron, d'Ydewalle et al. (2017) devise a new way to make it more effective.
authors.affiliation: Department of Genetics, Stanford University School of Medicine, Stanford, CA 94305, USA; Neurosciences Graduate Program, Stanford University School of Medicine, Stanford, CA 94305, USA.
authors.forename: Nicholas J
authors.initials: NJ
authors.lastname: Kramer
authors.affiliation: Department of Genetics, Stanford University School of Medicine, Stanford, CA 94305, USA. Electronic address: agitler@stanford.edu.
authors.forename: Aaron D
authors.initials: AD
authors.lastname: Gitler
mesh_terms: D015316:Genetic Therapy; D006801:Humans; D009134:Muscular Atrophy, Spinal / Q000235:genetics*
keywords: SMA; SMN; antisense; lncRNA; spinal muscular atrophy