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In world first, scientists use CRISPR to fix baby's unique genetic mutation

May 15, 2025
To our New York City area readers: Join us at our next STAT Locals event on June 5 to connect with others in the life sciences community (and get food and drink on us!).
Baby KJ with two of the researchers who treated him, Kiran Musunuru (left) and  Rebecca Ahrens-Nicklas.
Children's Hospital of Philadelphia

CRISPR is used in landmark treatment to correct genetic misspelling of a single patient

In a world first, scientists used CRISPR to fix a baby's unique genetic mutation. The report offers hope for personalized treatments of rare diseases.

By Jason Mast


Opinion: I was a Theranos whistleblower. Here's what I think Elizabeth Holmes is up to

STAT First Opinion essay: Tyler Shultz says he always knew Elizabeth Holmes would have a second act, "shocked it's starting while she's still behind bars."

By Tyler Shultz


STAT+ | FDA commissioner says new vaccine 'framework' for industry is coming within weeks

A top agency official is meeting with industry and FDA staff as he works on guidance clarifying the FDA's expectations for vaccines.

By Lizzy Lawrence



From left, STAT senior writer Matthew Herper speaks with Eric Rubin, Susan Desmond-Hellmann, and Georges Benjamin at the STAT Breakthrough Summit West in San Francisco on Wednesday.
Sarah Gonzales for STAT

'What wouldn't I be worried about?': Research leaders discuss threats to U.S. science

Scientists decried grant terminations and fear in academia as U.S. research faces mounting pressure, loss of global credibility

By Anil Oza


STAT+ | What to know about Summit Therapeutics' looming clinical trial readout

In this week's edition of "Adam's Biotech Scorecard," Adam offers a preview of what may be the most important study readout coming this summer.

By Adam Feuerstein


STAT+ | As AI in health care proliferates, so do legal questions concerning its use

Experts at STAT Breakthrough Summit West said developer disclaimers can be disingenuous, and keeping "a human in the loop" doesn't improve matters.

By Tara Bannow


Adobe

Opinion: Ensuring most-favored nation drug pricing doesn't sicken us

If the U.S. rationalizes how it pays for medicines, consumers, employers, and government payers would see fair prices.

By Darius Lakdawalla and Dana P. Goldman


Trump's drug-pricing plan and a potential Theranos 2.0

STAT reporters chat about Trump's drug-pricing plan, RFK Jr.'s congressional hearings, and a new blood-testing company.

By Elaine Chen and Adam Feuerstein


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