Chicken eggs will cure a serious hereditary disease

Ekaterina Korzeneva, N+1 

The American FDA regulator has allowed the use of genetically modified chickens as a source of raw materials for medicines in the treatment of one of the rare hereditary diseases. This is briefly reported by Nature (US government approves transgenic chicken).

The new line of animals is not intended for human consumption, but was created as a producer of the recombinant human enzyme sebelipase alpha. This molecule serves as an active component of the drug Kanuma, aimed at the treatment of a rare metabolic disorder – Wolman's disease. Pathology is caused by disorders of the LIPA gene – in the presence of damage to this gene in both chromosomes received by a child from parents, the disease manifests itself as an enzyme deficiency at the age of 3-6 months and often leads to death. In a healthy human body, this enzyme (acid lipase) is responsible for the breakdown of triglycerides and cholesterol esters inside cells. Its deficiency (partial absence) leads to the accumulation of fat in the liver, spleen and blood vessels, and in adulthood leads to fatty liver overgrowth and the development of fibrosis and cirrhosis, as well as heart failure. 

Thus, Kanuma, manufactured by Alexion Pharmaceuticals, is designed to make up for the deficiency of a natural enzyme in the body of patients. Before the advent of this drug, there was no treatment for Volman's disease: in case of illness, the condition of children was maintained by a special diet, and statins were used in adults – medications that reduce the amount of cholesterol in the blood.

The creation of genetically modified animals occurs as follows. The gene responsible for the production of the necessary protein (transgen) is embedded in a ring DNA molecule – a plasmid – in combination with a promoter and a terminator. These regulatory elements ensure the precise "on" and "off" of gene expression. The genetic construct is delivered to the embryonic stem cells of the future GM animal by microinjection. As a result of such manipulation, only a part of adult GM animals contains a transgen. In order for its integration to be stable and all animals in the offspring have this transgene, the first generation (F1) animals are crossed among themselves, then the presence of the transgene and the genotype of the animals are experimentally confirmed.

It is important to note that GM chicken has become the second genetically modified animal approved by the FDA in recent times. At the end of November, the agency allowed the first transgenic animal to be eaten, which was the AquAdvantage salmon. GM chicken has also become the second animal approved for the production of medicines: in February 2009, the FDA approved the use of goats, in whose milk an anticoagulant is synthesized, necessary for the treatment of various human blood clotting disorders.

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10.12.2015