METHODS FOR TREATMENT OF LYSOSOMAL STORAGE DISEASES USING BIPHOSPHONATES

摘要

Methods are disclosed for treatment of patients suffering from accumulation of a metabolite within macrophages, such as in lysosomal storage diseases. The methods comprise treating the patient with a macrophage depleting amount of a bisphosphonate compound, such that apoptosis of macrophages is induced and the metabolite is released into circulation so that the metabolites may be eliminated from the patient. The methods may further include administration of a gene therapy vector for the treatment of lysosomal storage diseases.