In the United States approved the intervention in human genes

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Photo: Photomall / Globallookpress.com

Management on sanitary inspection behind quality of foodstuff and medicines USA (FDA) first approved a drug intended for the treatment of hereditary amyloidosis transthyretin (hATTR). This publication reports Science Alert.

Parisian the drug blocks the synthesis of the mutated protein transthyretin (TTR), which is in normal form transports thyroid hormone Sirotin and vitamin a retinol. Defective protein contributes to the development of amyloidosis when tissue is the accumulation of the protein-polysaccharide complex — amyloid. Amyloid plaques contribute to the dysfunction of tissues and organs, including the heart.

The mechanism of suppression activity transthyretin based on RNA interference, when the expression of a gene is suppressed by using small RNA molecules (siRNAs). This process begins with cutting the long molecules of double-stranded foreign RNA (viruses belonging) for individual fragments. These parts are included in the RISC complex, which then combines with the complementary sequences of messenger RNA (mRNA) and trigger the cutting. This prevents the process of translation and protein synthesis, which was encoded in the mRNA.

Video, photo All from Russia.



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