Genistein (4', 5, 7-trihydroxyisoflavone or 5, 7-dihydroxy-3- (4-hydroxyphenyl)-4H-1-benzopyran-4-one) is a natural isoflavone of various biological activities. Recent studies indicated that this compound can be considered as a potential drug for treatment of some genetic diseases, exemplified by cystic fibrosis (CF) and mucopolysaccharidosis (MPS). Interestingly, molecular mechanisms by which genistein may be beneficial for patients suffering from these diseases are different. In CF, dysfunction of the CFTR gene causes a defect in the function of the transmembrane chloride pump, leading to a lack of movement of chloride in nose, sinuses, lungs, intestines, pancreas and sweat glands, and thus, to clinical manifestations of the disease. The most common mutations found in CF patients is DF508, and it appears that the function of the mutated gene product may be partially restored by genistein, which can interact with this protein, especially in combination with phenylbutyrate. MPS is a group of inherited metabolic disorders, caused by mutations leading to dysfunction of one of enzymes involved in degradation of glycosaminoglycans (GAGs) in lysosomes. Due to their impaired degradation, GAGs accumulate in cells of patients, which results in dysfunction of tissues and organs, including the heart, respiratory system, bones, joints and central nervous system. Genistein has been shown to act as an inhibitor of GAG synthesis due to its function of the inhibitor of kinase activity of epidermal growth factor receptor. Therefore, genistein, can be considered as a therapeutic for as complicated diseases as genetic disorders.

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1. SUBSTRATE DEPRIVATION THERAPY - ALTERNATIVE TREATMENT OF MUCOPOLYSACCHARIDOSES
2. EFFECT OF GENISTEIN SYNTHETIC DERIVATIVES ON REGULATION OF GLYCOSAMINOGLYCAN SYNTHESIS IN SANFILIPPO DISEASE (MUCOPOLYSACCHARIDOSIS TYPE III)
3. GENE EXPRESSION-TARGETED ISOFLAVONE THERAPY FOR MUCOPOLYSACCHARIDOSES