2019: the year gene therapy came of age

Washington, Dec 26 (AFP/APP): In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering — by editing her genome. Victoria Gray’s recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research — gene therapy. “I have hoped for a cure since I was about 11,” the 34-year-old told AFP in an email. “Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.” Over several weeks, Gray’s blood was drawn so doctors could get to the cause of her illness — stem cells from her bone marrow that were making deformed red blood cells. The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 — pronounced “Crisper” — a new tool informally known as molecular “scissors.” The genetically edited cells were transfused back into Gray’s veins and bone marrow. A month later, she was producing normal blood cells. Medics warn that caution is necessary but, theoretically, she has been cured. “This is one patient. … Continue reading 2019: the year gene therapy came of age