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New approach to gene therapy uses common pain reliever to fight against genetic diseases Researchers have developed a new approach to gene therapy that leans on the common pain reliever acetaminophen to force a variety of genetic diseases into remission. A paper published in Science Translational Medicine describes how the novel technique successfully treated the blood-clotting disorder hemophilia and the debilitating metabolic disease known as phenylketonuria, or PKU, in mice. The approach uses a benign lentivirus to both correct disease-causing mutations and to insert a new gene that makes liver cells immune to the potentially toxic effects of acetaminophen. The latter enables treated cells to survive and kills off untreated cells when they re repeatedly exposed to the over-the-counter medication that s also known by the brand name Tylenol. This allows the treated cells to multiply and creates a greater portion of liver cells being corrected.

United statesMarkus grompeEmily hendersonOregon health science university school of medicineScience translational medicineOregon healthScience university schoolஒன்றுபட்டது மாநிலங்களில்எமிலி ஹென்டர்சன்ஓரிகந் ஆரோக்கியம் அறிவியல் பல்கலைக்கழகம் பள்ளி ஆஃப் மருந்துஅறிவியல் மொழிபெயர்ப்பு மருந்துஓரிகந் ஆரோக்கியம்அறிவியல் பல்கலைக்கழகம் பள்ளி