What is in utero gene editing? скачать в хорошем качестве

What is in utero gene editing?

Recently approved gene therapies offer patients one-time, potentially curative treatments for genetic diseases such as sickle cell anemia and beta thalassemia. But “one-time” miracle solutions can often be multi-month affairs, require millions of dollars, and cause painful side effects. What if that doesn’t have to be the case? In utero gene editing, or prenatal somatic cell genome editing, envisions treating a fetus diagnosed with a genetic disease before birth, thereby preventing that entire protocol and the onset of symptoms in the first place. It would also challenge the need for the ethically fraught enterprise of embryo editing, as the treatment would only make edits in the DNA of the individual fetus — edits which would not be passed on in a heritable way. Watch this video to learn more about in utero gene editing, how it works, and why scientists believe it might be an advantageous approach to treating certain genetic diseases. More from STAT: Become a subscriber: https://www.statnews.com/stat-plus/ Watch Full Episodes:    / @statnews   Newsletters: https://www.statnews.com/signup/ STAT events: https://www.statnews.com/stat-events/ Facebook:   / statnews   Twitter:   / statnews   Instagram:   / statnews   Flipboard: rb.gy/3xnsxr STAT Reports: https://rb.gy/rexfwj STAT eBooks: https://rb.gy/eme3h5 ABOUT STAT: Founded in 2015, STAT is a global digital media brand that focuses on delivering fast, deep, and tough-minded journalism about the life sciences industries to over six million monthly site visitors and an additional 20 million readers on the Apple News app. STAT takes you inside academic labs, biotech boardrooms, and political backrooms, casting a critical eye on scientific discoveries, scrutinizing corporate strategies, and chronicling the roiling battles for talent, money, and market share. With an award-winning newsroom, STAT provides indispensable insights and exclusive stories on the technologies, personalities, power brokers, and political forces driving massive changes in the life sciences industry — and a revolution in human health.