A goat named Gluck against a hereditary disease

A genetically modified goat has been created to treat Gaucher's disease

Medical Xpress news: Goat to be cloned to treat rare genetic disorderBrazilian scientists have created a genetically modified goat that will produce milk with the enzyme glucocerebrosidase for the treatment of a genetic disease – Gaucher disease.

According to Medical Xpress with reference to the newspaper O Estado de Sao Paulo (Brasileiros produzem primeira cabra clonada e transgenica – VM), a goat named "Gluck", which became the first animal of its kind in South America, will start producing milk in about four months.

Gaucher disease (glucosylceramide lipidosis) is a rare hereditary disease that develops due to insufficient activity of the lysosomal enzyme glucocerebrosidase, responsible for the breakdown and utilization of the fatty substance glucocerebroside. In people with Gaucher disease, as a result of enzyme deficiency, this compound accumulates in the cells of the spleen, liver, kidneys, lungs, nervous system and bone marrow, making it difficult for them to function normally.

This disease manifests itself in the form of chronic fatigue, anemia, bruising, is characterized by a low level of platelets in the blood, an increase in the liver and spleen. As a rule, this lysosomal disease is treated with medications and periodic bone marrow transplants, but patients still experience pain and often have a poor long-term prognosis. Researchers estimate that Brazil, where more than 600 patients suffer from Gaucher's disease, spends about $113 million a year on their treatment.

The genetically modified Gluck goat (Gluca), on which scientists pin their hopes, was born on March 27. She is expected to have milk in about four months. "We will need to find out how much enzyme it will be able to produce, and how effective it will be," said researcher Luciana Bertolini from the University of Fortaleza. – It is cheaper to feed goats to produce the enzyme than to grow cell lines. At the same time, the purification of the protein necessary for the treatment of Gaucher's disease is generally the same."

If everything goes as scientists plan, and the milk of a genetically modified animal contains a high percentage of the enzyme glucocerebrosidase, the researchers will clone the goat to get the necessary protein on a larger scale.

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