The major neurodegenerative diseases, such as Alzheimer's, Parkinson's, and Huntington's disease, are caused by the misfolding of specific proteins.  With its traditional strength in the biophysical and molecular analysis of proteins, Boston Biomedical Research Institute is strongly positioned to analyze the molecular basis of these disorders to discover ways to correct the folding defects.  These studies are aided by the availability at the Institute of cellular models of Huntington's based on yeast and animal cells and of animal models for both Huntington's and Parkinson's disease.  BBRI researchers have the ability to monitor mouse behavior in order to evaluate possible therapeutics and to understand how body structures, disease and other processes contribute to the body tissue loss or dysfunction. A better understanding of these will lead to the successful treatment of Huntington's, Parkinson's, Alzheimer’s and other neurological diseases.

Congenital and adult forms of muscular dystrophy are caused by genetic defects that lead to severe atrophy of muscle tissue.  The onset of the disease and the types of muscle affected are determined by the underlying genetic mutations, and no single treatment is therefore possible.  Institute scientists are studying several different muscular dystrophies to identify their particular molecular defects and possible modes of intervention. The Wellstone Muscular Dystrophy Cooperative Research Center has been established at BBRI to study facioscapulohumeral muscular dystrophy (FSHD) and other muscle diseases and provide a cell repository as a resource for the study of neuromuscular diseases. By learning how muscles grow and deteriorate and the role that specific proteins play in these processes, Boston Biomedical’s Regenerative Biology team will discover how to promote muscle regeneration and develop treatments for neuromuscular diseases.

The therapeutic agents currently being explored by Institute scientists are small molecule drugs against Huntington's, hormonal intervention for Parkinson's, and a vaccine strategy against Alzheimer's disease.