title: The potential of sarcospan in adhesion complex replacement therapeutics for the treatment of muscular dystrophy.
abstract: Three adhesion complexes span the sarcolemma and facilitate critical connections between the extracellular matrix and the actin cytoskeleton: the dystrophin- and utrophin-glycoprotein complexes and α7β1 integrin. Loss of individual protein components results in a loss of the entire protein complex and muscular dystrophy. Muscular dystrophy is a progressive, lethal wasting disease characterized by repetitive cycles of myofiber degeneration and regeneration. Protein-replacement therapy offers a promising approach for the treatment of muscular dystrophy. Recently, we demonstrated that sarcospan facilitates protein-protein interactions amongst the adhesion complexes and is an important potential therapeutic target. Here, we review current protein-replacement strategies, discuss the potential benefits of sarcospan expression, and identify important experiments that must be addressed for sarcospan to move to the clinic.
authors.affiliation: Department of Integrative Biology and Physiology, University of California, Los Angeles, CA 90095, USA.
authors.forename: Jamie L
authors.initials: JL
authors.lastname: Marshall
authors.forename: Yukwah
authors.initials: Y
authors.lastname: Kwok
authors.forename: Brian J
authors.initials: BJ
authors.lastname: McMorran
authors.forename: Linda G
authors.initials: LG
authors.lastname: Baum
authors.forename: Rachelle H
authors.initials: RH
authors.lastname: Crosbie-Watson
mesh_terms: D000818:Animals; D002352:Carrier Proteins / Q000378:metabolism*; D016189:Dystrophin / Q000378:metabolism*; D006801:Humans; D016023:Integrins / Q000378:metabolism*; D008565:Membrane Proteins / Q000378:metabolism*; D009136:Muscular Dystrophies / Q000378:metabolism / Q000628:therapy*; D009363:Neoplasm Proteins / Q000378:metabolism*; D012508:Sarcolemma / Q000378:metabolism*; D015398:Signal Transduction; D049411:Utrophin / Q000378:metabolism*
chemical_list: D002352:Carrier Proteins; D016189:Dystrophin; D016023:Integrins; D008565:Membrane Proteins; D009363:Neoplasm Proteins; C109646:SSPN protein, human; D049411:Utrophin; C082908:integrin alpha7beta1
keywords: Duchenne; dystrophin; integrin; laminin-binding; mdx; muscle; muscular dystrophy; sarcolemma; sarcospan; utrophin