Related Experiment Video
Updated: Mar 9, 2026

Delivery of Therapeutic Agents Through Intracerebroventricular ICV and Intravenous IV Injection in Mice
Published on: October 3, 2011
Raise the Roof: Boosting the Efficacy of a Spinal Muscular Atrophy Therapy
Nicholas J Kramer1, Aaron D Gitler2
1Department of Genetics, Stanford University School of Medicine, Stanford, CA 94305, USA; Neurosciences Graduate Program, Stanford University School of Medicine, Stanford, CA 94305, USA.
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.
More Related Videos
10:57Intramuscular Injections Along the Motor End Plates: A Minimally Invasive Approach to Shuttle Tracers Directly into Motor Neurons
Published on: July 13, 2015
07:02Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
Published on: May 11, 2018
Related Concept Videos
Myasthenia Gravis: Overview and Treatment
These antibodies interfere with the function of the nicotinic receptors in three ways: by binding to the receptor and disrupting acetylcholine binding; by causing cross-linking of receptors which...
Myasthenia Gravis: Diagnostic Tests
The edrophonium test is a diagnostic tool for myasthenia gravis. It involves...
Satellite Stem Cells and Muscular Dystrophy
Skeletal Muscle Relaxants: Therapeutic Uses