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Development of an Osteoprotegerin Gene Therapy to Increase Bone Density

An orally administered gene therapy using macrophage targeted yeast cell wall particles containing DNA encoding human osteoprotegerin, OPG, is being developed to increase bone density and quality by reducing the extent of osteoclast mediated bone resorption. Low bone mass such as osteoporosis is a major health risk for millions of women and men, predisposing individuals to bone fractures, as well as sequelae including stroke and an accelerated decrease in quality of life. Administration of OPG improves the clinical manifestations of bone loss, but current formulations of OPG proteins must be recombinantly produced and intravenously administered. It is anticipated that the improved delivery of OPG to macrophages and osteoclasts in bone by this gene therapy approach will produce increased bone density as a result of reduced bone resorption. The proposed studies will assess the in-vivo impact of this therapeutic approach to improve the skeletal manifestations of low bone density and excessive bone resorption.

Development of an Orally Administered Gene Therapy for Gaucher Disease

An orally administered gene therapy is being been developed as a next generation therapeutic to restore normal enzymatic activity in Gaucher disease, the most common lysosomal storage disorder. This approach uses yeast cell wall particles containing DNA or RNA encoding human glucocerebrosidase to restore normal enzyme in-vivo in Gaucher mice. The skeletal and central nervous system complications of Gaucher disease still present an enormous challenge for current enzyme and gene replacement therapies. Despite the successes of gene therapy strategies in animal models, the clinical trials conducted to date have generally resulted in low levels of gene expression. Studies will determine the extent of improvement in enzyme levels in macrophages and tissues of treated Gaucher mice, the extent of reversal of lipid storage and tissue pathology and impact on survival. We anticipate that this approach will provide improved delivery of human glucocerebrosidase to many tissues, including bone. If macrophages containing human glucocerebrosidase migrate into brain, the resulting increased enzyme levels could provide clinical benefit for the neurological manifestations of Gaucher disease. The successful development of this therapeutic strategy should provide a safer, more efficient and cost effective treatment for patients with Gaucher disease, as well as providing a prototype of therapy to benefit those having a wide range of other lysosomal diseases.

Identification of Gene Mutations Causing Human Disease

Genetic studies are carried out to obtain a better understanding of the molecular basis of human diseases by identifying correlations between molecular variations and clinical manifestations. Examples of clinical research projects with molecular components include: psychiatric disorders (manic-depressive illness; schizophrenia); Gaucher disease and other inherited metabolic disorders; and, Ellis van-Creveld dwarfism.

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