Promising gene therapy delivers treatment directly to brain
When Rylae-Ann Poulin was a year old, she didn’t crawl or babble like other kids her age. A rare genetic disorder kept her from even lifting her head. Her parents …
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Study offers a genetic explanation for why some drugs trigger a deadly brain disease
Medicines that reshape or tamp down immune responses may be life-changing for patients with cancer and autoimmune disorders, but in some cases they can awaken a dormant virus and unleash …
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Huge U.S. study starts sharing gene findings with volunteers
More than 155,000 Americans who shared their DNA for science are about to learn something in return: Do they have some particularly worrisome genes? It’s part of a massive project to …
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‘Make People Better’: The CRISPR babies story you probably don’t know, and how it was filmed in real-time
In May of 2017, Samira Kiani found herself in a San Diego hotel ballroom surrounded by some of the CRISPR field’s brightest shining stars. Jennifer Doudna, George Church, and others …
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Revenge of the gaslit patients: Now, as scientists, they’re tackling Ehlers-Danlos syndromes
Type “Ehlers-Danlos syndromes” into a search engine, and multiple suggestions pop up. Is Ehlers-Danlos an autoimmune disease? Is EDS hereditary? Rare? Then, the algorithm might suggest: Is Ehlers-Danlos syndrome a …
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On the long road to treating Huntington’s genetic stutter, scientists return to overlooked clues
In the spring of 1981, a geneticist from Indianapolis and a neurologist from Chicago got in a car and for three days they crisscrossed the yellowing cornfields of Iowa. Every …
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After three years in prison, ‘CRISPR babies’ scientist is attempting a comeback
He Jiankui, the Chinese biophysicist who created the first gene-edited children, had been quiet since completing a three-year prison sentence in April, leaving many to wonder whether he had plans …
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Perfection is too high a bar for CRISPR treatments, says STAT Biomedical Innovation Award winner David Liu
BOSTON — In science journalism, there’s a well-worn technique reporters reach for when they’re interviewing a researcher and the technical material starts whizzing over their heads. “OK, explain it again, …
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In a first, a fatal enzyme deficiency is treated in the womb
To protect this child from the same genetic disease that killed two older siblings, treating her as soon as she was born might only work so well, the doctors knew. …
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