FDA Approves New Gene-Silencing Drug

The Food and Drug Administration has recently approved the use of a breakthrough treatment involving a drug that utilizes double stranded RNA to silence specific genes in a patient’s body. The process, which also occurs naturally, is called RNA interference.

This special type of RNA, or ribonucleic acid, is nearly identical to DNA structurally. However, DNA contains adenine, thymine, guanine, and cytosine as its four bases, while RNA replaces thymine with uracil. RNA is primarily used to build proteins, while DNA replicates and stores genetic information.

The drug, patisiran, which was approved August 10th, targets a rare hereditary disease called hereditary transthyretin amyloidosis, or hATTR. It helps patients of the disease who have neurological impairment by silencing a mutated gene that is responsible for their symptoms. It does this by using special segments of synthetic RNA delivered specifically to the liver to artificially manipulate the activity of normal genes. RNA interference is also being researched as a possible treatment for HIV and may save countless lives in the next few decades.

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