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Gene-editing treatments have arrived

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Fri, Dec 8, 2023 09:17 PM

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Plus more health news | Email not displaying correctly? By Alice Park Senior Health Correspondent Af

Plus more health news | Email not displaying correctly? [View it in your browser.]( [Health Matters]( [The FDA just approved the very first CRISPR-based gene-editing treatment, for sickle-cell anemia]( By Alice Park Senior Health Correspondent After decades without many options for treating their disease, sickle cell patients now have two cutting-edge therapies with the potential to cure the illness. On Dec. 8, the U.S. Food and Drug Administration approved two gene therapies to treat sickle cell: Lyfgenia, from Bluebird Bio, and Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics. Both new treatments work by helping patients' bodies make healthier and more durable forms of hemoglobin, hopefully for the rest of their lives. Both also require nearly a year of procedures, including a bone marrow transplant and high-dose chemotherapy to remove the diseased cells and make room for the modified ones that can produce normal hemoglobin. Studies show that nearly all patients receiving either treatment are able to avoid the disease's characteristic periodic episodes of extreme pain that occur when sickled cells clump together and block small blood vessels, Casgevy is especially groundbreaking, as it is the first CRISPR-based treatment to be approved in the U.S., and could open the door for other such therapies for different genetic diseases. It works kind of like "removing the stop sign and allowing traffic—in this case, [healthy] fetal hemoglobin—to go forward down the road,” says Dr. Sharl Azar, medical director of the comprehensive sickle cell disease treatment center at Massachusetts General Hospital. The FDA will require both companies to continue studying patients for 15 years to track any long term side effects. [READ MORE]( Share This Story What Else to Read [6 Ways to Set Boundaries at Work—Even When It's Uncomfortable]( By Angela Haupt Yes, even in this economy. [Read More »]( [The Obvious Solution to Loss-and-Damage Fund Distribution]( By Yolande Wright How to effectively distribute climate loss-and-damage funds is a key question at the COP28 meeting in Dubai. Yolande Wright has the answer. [Read More »]( [The Case for Mediocrity]( By Jamie Ducharme Why "good enough" really is good enough. [Read More »]( [Column: No AI Can Learn the Art of Medicine]( By Robert Glatter, Peter Papadakos, and Yash Shah Practicing medicine is an art and AI threatens the core values that doctors use to treat patients. [Read More »]( [Column: Believing Myths About Aging Makes Growing Old Worse]( By Steven Kotler Believing the myths about aging actually makes us age faster. [Read More »]( AN EXPERT VOICE "It’s a wonderful time to declutter. Around the holidays, we tend to accumulate more. What stuff do you need, and what do you need to release? You might need more efficiency in your life—and by decluttering, you’ll improve your mental health." —therapist Nedra Glover Tawwab, author of Set Boundaries, Find Peace: A Guide to Reclaiming Yourself If you were forwarded this and want to sign up to receive it daily, [click here](. Today's newsletter was written by Alice Park and Angela Haupt, and edited by Elijah Wolfson. [Want more from TIME? Sign up for our other newsletters.]( [Subscribe to TIME]( TIME may receive compensation for some links to products and services in this email. Offers may be subject to change without notice. Connect with TIME via [Facebook]( | [Twitter]( | [Newsletters]( [UNSUBSCRIBE]( [PRIVACY POLICY]( [YOUR CALIFORNIA PRIVACY RIGHTS]( TIME Customer Service, P.O. Box 37508, Boone, IA 50037-0508 Questions? Contact health@time.com Copyright © 2023 TIME USA, LLC. All rights reserved.

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