掀起屋顶：提高脊髓性肌萎缩症疗法的疗效。

Raise the Roof: Boosting the Efficacy of a Spinal Muscular Atrophy Therapy.

机构信息

Department of Genetics, Stanford University School of Medicine, Stanford, CA 94305, USA; Neurosciences Graduate Program, Stanford University School of Medicine, Stanford, CA 94305, USA.

Department of Genetics, Stanford University School of Medicine, Stanford, CA 94305, USA.

出版信息

Neuron. 2017 Jan 4;93(1):3-5. doi: 10.1016/j.neuron.2016.12.029.

DOI:10.1016/j.neuron.2016.12.029

PMID:28056344

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.

摘要

脊髓性肌萎缩症是婴儿最常见的遗传杀手。一种疗法在临床上显示出前景，但它的疗效可能存在局限。在本期《神经元》杂志上，d'Ydewalle 等人（2017）设计了一种使它更有效的新方法。