gene editing

CRISPR base editing gives measure of hope to people with muscular dystrophy

Next year, a neurologist will test CRISPR base editing in a trial of five people with muscular dystrophy to see if their muscles accept corrected cells and whether they multiply and take over the function of damaged cells.

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When Martin Weber climbs the steps to his apartment on the fifth floor in Munich, an attentive observer might notice that he walks a little unevenly. “That’s because my calf muscles were the first to lose strength,” Weber explains.

About three years ago, the now 19-year-old university student realized that he suddenly had trouble keeping up with his track team at school. At tennis tournaments, he seemed to lose stamina after the first hour. “But it was still within the norm,” he says. “So it took a while before I noticed something was seriously wrong.” A blood test showed highly elevated liver markers. His parents feared he had liver cancer until a week-long hospital visit and scores of tests led to a diagnosis: hereditary limb-girdle muscular dystrophy, an incurable genetic illness that causes muscles to deteriorate.

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Michaela Haas
Michaela Haas, PhD, is an award-winning reporter and author, most recently of Bouncing Forward: The Art and Science of Cultivating Resilience (Atria). Her work has been published in the New York Times, Mother Jones, the Huffington Post, and numerous other media. Find her at www.MichaelaHaas.com and Twitter @MichaelaHaas!
Researchers Get Closer to Gene Editing Treatment for Cardiovascular Disease

Scientists are making progress to create a one-time therapy that would permanently lower LDL cholesterol to prevent heart attacks caused by high LDL.

Verve Therapeutics

Later this year, Verve Therapeutics of Cambridge, Ma., will initiate Phase 1 clinical trials to test VERVE-101, a new medication that, if successful, will employ gene editing to significantly reduce low-density lipoprotein cholesterol, or LDL.

LDL is sometimes referred to as the “bad” cholesterol because it collects in the walls of blood vessels, and high levels can increase chances of a heart attack, cardiovascular disease or stroke. There are approximately 600,000 heart attacks per year due to blood cholesterol damage in the United States, and heart disease is the number one cause of death in the world. According to the CDC, a 10 percent decrease in total blood cholesterol levels can reduce the incidence of heart disease by as much as 30 percent.

Verve’s Founder and CEO, Sekar Kathiresan, spent two decades studying the genetic basis for heart attacks while serving as a professor of medicine at Harvard Medical School. His research led to two critical insights.

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Christopher Johnston
Christopher Johnston has published more than 3,500 articles in publications including American Theatre, Christian Science Monitor, History Magazine, and Scientific American. His book, Shattering Silences: Strategies to Prevent Sexual Assault, Heal Survivors, and Bring Assailants to Justice (Skyhorse) was published in May 2018. He is a member of the Board of the American Society of Journalists and Authors.
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Science Has Given Us the Power to Undermine Nature's Deadliest Creature: Should We Use It?

The Aedes aegypti mosquito, which can carry devastating diseases, was recently engineered by a biotech company to have a genetic "kill switch" intended to crash the local population in the Florida Keys.

Adobe

Lurking among the swaying palm trees, sugary sands and azure waters of the Florida Keys is the most dangerous animal on earth: the mosquito.

While there are thousands of varieties of mosquitoes, only a small percentage of them are responsible for causing disease. One of the leading culprits is Aedes aegypti, which thrives in the warm standing waters of South Florida, Central America and other tropical climes, and carries the viruses that cause yellow fever, dengue, chikungunya and Zika.

Dengue, a leading cause of death in many Asian and Latin American countries, causes bleeding and pain so severe that it's referred to as "breakbone fever." Chikungunya and yellow fever can both be fatal, and Zika, when contracted by a pregnant woman, can infect her fetus and cause devastating birth defects, including a condition called microcephaly. Babies born with this condition have abnormally small heads and lack proper brain development, which leads to profound, lifelong disabilities.

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Eve Herold

Eve Herold is a science writer specializing in issues at the intersection of science and society. She has written and spoken extensively about stem cell research and regenerative medicine and the social and bioethical aspects of leading-edge medicine. Her 2007 book, Stem Cell Wars, was awarded a Commendation in Popular Medicine by the British Medical Association. Her 2016 book, Beyond Human, has been nominated for the Kirkus Prize in Nonfiction, and a forthcoming book, Robots and the Women Who Love Them, will be released in 2019.

How mRNA Could Revolutionize Medicine
Photo by the National Cancer Institute on Unsplash

In November 2020, messenger RNA catapulted into the public consciousness when the first COVID-19 vaccines were authorized for emergency use. Around the same time, an equally groundbreaking yet relatively unheralded application of mRNA technology was taking place at a London hospital.

Over the past two decades, there's been increasing interest in harnessing mRNA — molecules present in all of our cells that act like digital tape recorders, copying instructions from DNA in the cell nucleus and carrying them to the protein-making structures — to create a whole new class of therapeutics.

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David Cox
David Cox is a science and health writer based in the UK. He has a PhD in neuroscience from the University of Cambridge and has written for newspapers and broadcasters worldwide including BBC News, New York Times, and The Guardian. You can follow him on Twitter @DrDavidACox.