Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of Concept.

ABSTRACT:

Objective

Preclinical therapies that restore survival motor neuron (SMN) protein levels can dramatically extend survival in spinal muscular atrophy (SMA) mouse models. Biomarkers are needed to effectively translate these promising therapies to clinical trials. Our objective was to investigate electrophysiological biomarkers of compound muscle action potential (CMAP), motor unit number estimation (MUNE) and electromyography (EMG) using an SMA mouse model.

Methods

Sciatic CMAP, MUNE, and EMG were obtained in SMNΔ7 mice at ages 3-13 days and at 21 days in mice with SMN selectively reduced in motor neurons (ChAT^Cre ). To investigate these measures as biomarkers of treatment response, measurements were obtained in SMNΔ7 mice treated with antisense oligonucleotid

SUBMITTER: Arnold WD 

PROVIDER: S-EPMC3914317 | biostudies-literature | 2014 Jan

REPOSITORIES: biostudies-literature