GENE THERAPY TO TREAT HYPERAMMONEMIA IN HEPATIC ENCEPHALOPATHY AND OTHER LIVER DISEASES.

摘要

The present disclosure is related to the production of a new drug for the treatment of Hepatic Encephalopathy (HE), which consists of recombinant viruses, in particular baculoviruses, which over-expresses one or more of the ammonia metabolism enzymes including Glutamine synthetase (GS), in various tissues of the patient and with the purpose of reducing the acute hyperammonemia and relief the signs and symptoms of HE. The recombinant baculovirus Bac-GS developed in the present invention expresses the RNAm and the GS and, as a result, reduces the levels of ammonium in the in-vitro and in-vivo medium. For this purpose, a new in-vitro hyperammonemia model was generated. This new drug called Bac-GS can be used to reduce acute hyperammonemia in patients with urea cycle disorders and other diseases with high ammonia levels, such as: acute and chronic liver failure, hepatocellular carcinoma and diseases related to neuropsychiatric disorders.