Hi I’m JK, the first baby treated with a personalized CRISPR therapy

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The announcement was made yesterday at the annual meeting of the American Society of Gene & Cell Therapy and simultaneously published in the New England Journal of Medicine. I wrote about it for the Italian edition of Scientific American, but this story is worth seeing and reading.
PS: Before KJ was treated for his metabolic disorder, there was the case of Terry Horgan, who had muscular dystrophy. Unfortunately, the personalized treatment for him came too late, and he did not survive an adverse reaction. So yes, we can say that KJ represents a first: the first time genome editing was used early, rapidly, and tailored enough to truly offer hope for a happy ending.

CRISPR pork and the challenge of animal welfare

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Classic genetic modification has met the most resistance in the livestock sector, but soon, American consumers will be able to eat meat from the first gene-edited animals: pigs immune to the PRRS virus.

BBQ ribs, pulled pork, crispy bacon. In the near future, those visiting the U.S. will likely have the opportunity to taste classic American dishes in a genetically edited form. The Food and Drug Administration has, in fact, approved the first pigs whose genome has been edited using CRISPR to make them resistant to a serious viral disease: porcine reproductive and respiratory syndrome (PRRS).

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Mendel’s 7 genes

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Can you never remember all seven of Snow White’s dwarfs? Then try the seven traits studied in peas by Mendel. Smooth or wrinkled seeds, yellow or green seeds, white or purple flowers… I used to stop there until I read about the latest study in Nature. Joining forces, the John Innes Centre in Norwich, UK, and the Institute of Agricultural Genomics in Shenzhen, China, have identified Mendel’s remaining genes—solving a conundrum in the history of science and laying the groundwork for a leap forward in the genetic improvement of a food crop that is nutritious but too often overlooked by geneticists.

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Crying dire wolf

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The dire wolves “genetically resurrected” by the U.S.-based company Colossal have impressed many with their beauty but have also scandalized others. Among the latter are commentators criticizing the media hype, and naturalists worried that a few partially “de-extinct” animals may attract more attention than the long list of species currently at risk of extinction due to climate change and other human actions. It doesn’t help that the dire wolves’ comeback has no foundation in published scientific data—no peer-reviewed articles, no preprints yet, only two extensive reportages and some YouTube videos. There is certainly room for controversy. However, if we only listen to the critical voices, we risk losing sight of some key facts.

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Breakthrough Prize 2025: Jodie Foster explaining gene editing and more

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Gene editing pioneer David Liu received the Breakthrough Prize from the hands of Jodie Foster and Lily Collins — but the biggest applause went to young CAR-T patient Alyssa Tapley.

The movie stars in the audience in Santa Monica on April 12 were easy to spot: Brad Pitt, Margot Robbie, Sean Penn, and others. But it’s rare for a scientist to become a celebrity beyond academic circles. This role reversal happens just one day a year, when the Breakthrough Prize is celebrated in California. Richer than the Nobel (the prizes are worth three times as much) and steeped in glamour, the event honors the stars of science with the help of Hollywood and technofinance.

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Agrobiodiversity, pangenomes and the future of food

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Comparing commonly grown species and native varieties is a winning strategy for making the former more resilient and the latter more productive.

You all know tomatoes and potatoes. African eggplants, maybe not—but when ripe, they turn red just like tomatoes. The lulo, for its part, is an orange fruit with citrusy notes, which is why in Ecuador it’s called naranjilla, or “little orange.” The Andean pepino, on the other hand, has juicy flesh that makes it resemble a melon. Their sizes, colors, and flavors may vary, but all of these plants belong to the same taxonomic group. In fact, they represent some of the species sequenced to produce a remarkable collection of related genomes—remarkable because it aims to span the entire Solanum genus.

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CRISPR News – A Trio of Firsts

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Many interesting papers have been published recently; here are our top three picks. They cover an innovative gene therapy trial, a new experimental approach for oncology, and the development of novel tools to map gene enhancers.

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Is RNA unwelcome? Let’s hope it’s a misunderstanding

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source: Nature Biotechnology

Rumor has it that RNA has fallen out of favor politically, presumably as a key molecule for vaccines against Covid, making it an unwelcome symbol to the U.S. administration in the era of Robert F. Kennedy Jr. and Donald Trump. According to reports, U.S. researchers have been advised to specifically remove references to messenger RNA from research projects competing for public funding.

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