In The United States, They Approved The Intervention In Human Genes - Alternative View

In The United States, They Approved The Intervention In Human Genes - Alternative View
In The United States, They Approved The Intervention In Human Genes - Alternative View

Video: In The United States, They Approved The Intervention In Human Genes - Alternative View

Video: In The United States, They Approved The Intervention In Human Genes - Alternative View
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The US Food and Drug Administration (FDA) first approved a drug for the treatment of hereditary transthyretin amyloidosis (hATTR). This is reported by Science Alert.

The drug patisiran blocks the synthesis of the mutated protein transthyretin (TTR), which in normal form carries the thyroid hormone tyrotin and the vitamin retinol. A defective protein contributes to the development of amyloidosis, when the accumulation of a protein-polysaccharide complex - amyloid - occurs in the tissues. Amyloid plaques contribute to tissue and organ dysfunction, including the heart.

The mechanism of suppression of transthyretin activity is based on RNA interference, when the expression of a particular gene is suppressed using small RNA molecules (siRNA). This process begins with the cutting of long foreign double-stranded RNA molecules (belonging to viruses) into separate fragments. These parts are included in the RISC complex, which then binds to complementary messenger RNA (mRNA) sequences and activates their cleavage. This prevents the translation process and the synthesis of the protein that has been encoded in the mRNA.

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