We found a match
Your institution may have access to this item. Find your institution then sign in to continue.
- Title
Utility of survival motor neuron ELISA for spinal muscular atrophy clinical and preclinical analyses.
- Authors
Kobayashi, Dione T; Olson, Rory J; Sly, Laurel; Swanson, Chad J; Chung, Brett; Naryshkin, Nikolai; Narasimhan, Jana; Bhattacharyya, Anuradha; Mullenix, Michael; Chen, Karen S
- Abstract
Genetic defects leading to the reduction of the survival motor neuron protein (SMN) are a causal factor for Spinal Muscular Atrophy (SMA). While there are a number of therapies under evaluation as potential treatments for SMA, there is a critical lack of a biomarker method for assessing efficacy of therapeutic interventions, particularly those targeting upregulation of SMN protein levels. Towards this end we have engaged in developing an immunoassay capable of accurately measuring SMN protein levels in blood, specifically in peripheral blood mononuclear cells (PBMCs), as a tool for validating SMN protein as a biomarker in SMA.
- Publication
PloS one, 2011, Vol 6, Issue 8, pe24269
- ISSN
1932-6203
- Publication type
Journal Article
- DOI
10.1371/journal.pone.0024269