Genistein: a natural isoflavone with a potential for treatment of genetic diseases

These results indicate that genistein may be an even more potent drug in the treatment of CF than suggested previously, as it may enhance expression, proper localization and activity of the mutant protein

Grzegorz Węgrzyn; Joanna Jakóbkiewicz-Banecka; Magdalena Gabig-Cimińska; Ewa Piotrowska; Magdalena Narajczyk; Anna Kloska; Marcelina Malinowska; Dariusz Dziedzic; Izabela Gołębiewska; Marta Moskot; Alicja Węgrzyn

2010

Scholarcy highlights

  • Genistein is a common name of 5,7-dihydroxy3-(4-hydroxyphenyl)-4H-1-benzopyran-4-one
  • Moderate concentrations of genistein augmented CFTR maturation and increased its localization to the cell surface 25. These results indicate that genistein may be an even more potent drug in the treatment of CF than suggested previously, as it may enhance expression, proper localization and activity of the mutant protein
  • Genistein affected EGFR-catalysed phosphorylation efficiency 34. These results, together with the finding that efficiency of GAG synthesis may significantly influence the severity of MPS diseases 35, support the validity of the name proposed for the therapy based on the use of genistein in MPS: GET IT
  • A recent pilot clinical study with ten patients suffering from MPS IIIA and MPS IIIB indicated that genistin-rich soya isoflavone extract, administered orally for 12 months at the dose corresponding to 5 mg of genistein per 1 kg of body weight daily, caused a statistically significant improvement in all parameters tested 36
  • Since accumulation of GAGs occurs in some other LSDs 2627, it appears that further studies on the mechanisms of LSDs and activities of genistein and other flavonoids should be important in development of novel therapeutic procedures for as yet non-treatable genetic diseases
  • Since in the pilot clinical study with GET IT for MPS III a soya isoflavone extract was used rather than pure genistein 36, a question appeared as to whether a natural source of this isoflavone or a synthetic compound should be the final form of the drug
  • Molecular mechanisms of action of genistein in these two cases are different, multiple biological activities of this isoflavone make it an attractive compound when a safe and effective treatment of a complicated disease is considered

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