Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy
Abstract
Duchenne muscular dystrophy (DMD) is a rare and devastating muscle disease caused by mutations in the X-linked DMD gene (which encodes the dystrophin protein). Serum biomarkers hold significant potential as objective phenotypic measures of DMD disease state, as well as potential measures of pharmacological effects of and response to therapeutic interventions. Here we describe a proteomics approach to determine serum levels of 1,125 proteins in 93 DMD patients and 45 controls. The study identified 44 biomarkers that differed significantly between patients and controls. These data are being made available to DMD researchers and clinicians to accelerate the search for new diagnostic, prognostic, and therapeutic approaches.
Document Details
- Document Type
- Pub Defense Publication
- Publication Date
- May 26, 2015
- Source ID
- 10.1073/pnas.1507719112
Entities
People
- Angela Lorts
- Bob Mehler
- Britta Singer
- Craig M. McDonald
- David Sterling
- Edward Brody
- Eric Hoffman
- Erik Henricson
- Fintan Steele
- H. Lee Sweeney
- Heather Gordish-Dressman
- Jean K Mah
- Larry Gold
- Lauren Hache
- Linda Cripe
- Malti Nikrad
- Pat Furlong
- Paula R. Clemens
- Robert Kirk Delisle
- Sally Nelson
- Steve Williams
- Yetrib Hathout
- Yvonne Monique Kobayashi
Organizations
- Children's National Hospital
- Heart Institute
- Indiana University School of Medicine
- National Institutes of Health
- Ohio State University
- Parent Project Muscular Dystrophy
- United States Department of Defense
- United States Department of Education
- United States Department of Veterans Affairs
- University of Calgary
- University of California, Davis, School of Medicine
- University of Colorado Denver
- University of Florida
- University of Pittsburgh