Protein Therapy for Muscular Dystrophy and Other Muscle Diseases
AuthorVan Ry, Pam Marie
AdvisorBurkin, Dean J
Biochemistry and Molecular Biology
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Despite the exponential advancements in understanding the underlying cause of muscular diseases, there has been little progress in developing an effective treatment which results in the quality of life treatment options desperately needed for patients and families. The muscular dystrophies are a group of progressive degenerative muscle wasting diseases that vary in age of onset, phenotype, cause, severity and life span. Over 90 years ago protein therapy was used to treat diabetes. Today there are over 130 proteins or peptides approved by the US Food and Drug Administration (FDA) for clinical use in almost every field of medicine. Protein therapies have proven to be advantageous and effective since insulin was first used. This work shows that laminin-111 therapy can increase regeneration of skeletal muscle and encourages activation and formation of de novo skeletal muscle. Recombinant Galectin-1 therapy in dystrophic muscle was able to stabilize myofibers functionally through increases in members of the utrophin glycoprotein complex and α7β1 integrin. This thesis demonstrates that protein replacement therapy is a viable treatment option for muscle disease and will translate into measurable quality of life changes for patients and families.