CRISPR: hitting the genetic bullseye

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Imagine throwing a trillion darts and having every single one hit the bullseye. Achieving this level of precision in gene editing would require highly intelligent delivery of the molecular machinery for DNA repair (CRISPR or one of its variants) into patients’ bodies, reaching only defective cells while bypassing healthy tissues. The benefits would be substantial: maximum therapeutic efficiency, zero waste, and reduced risks in terms of toxicity, immunogenicity, and unwanted mutations. How can such precise targeting be achieved? By acting on multiple levels, explain Jennifer Doudna and three researchers from her Innovative Genomics Institute. See their review article, Targeted delivery of genome editors in vivo in Nature Biotechnology.

ISAAA: a window on plant gene editing

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The blog also aims to highlight authoritative sources to keep readers up to date. ISAAA is an international non-profit organization dedicated to documenting applications of agricultural biotechnology and providing accurate information. We are pleased to recommend its supplement focused on gene editing. The latest issue covers a high-yield rice variety ready for cultivation in India, another drought-resistant rice developed in Indian laboratories, a genetically modified potato designed to reduce waste in Chile, and, finally, a scanner developed in Berkeley to identify gene switches in plants. Enjoy reading!

Gene editing’s new bet on PKU

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Jennifer Doudna and Fyodor Urnov have founded a company that will take on the challenge of editing rare diseases, starting with phenylketonuria.

The problem is well known: many diseases are theoretically treatable by correcting the corresponding genetic defects with the help of CRISPR tools, but doing so risks being difficult or even impossible because of regulatory rigidity and economic unsustainability. The case of the first newborn treated with a bespoke therapy developed in record time (KJ Muldoon) was a splendid proof of principle. But it left many parents of children with rare diseases asking: when will it be our turn? At the same time, specialists have long been asking: will investors return to viewing gene editing as a profitable approach worth investing in?

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A fresh graphic take on gene editing

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Tired of illustrating articles and PowerPoint presentations on genome editing with the usual genetic scissors and Swiss Army knives? You might not know that the Innovative Genomics Institute, founded by CRISPR inventor Jennifer Doudna, offers images created by its graphic designers free of charge for educational and non-commercial use. Take a look at the Science × Art section of their website and their illustrated glossary.

A new atlas for plant genomes

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As we become increasingly able to reshape genomes, the need to organize information in efficient and accessible ways continues to grow. More than a thousand plant genomes have now been sequenced, with the pace accelerating, so it is good news that the plant genetics community can rely on PubPlant, a new interactive, updatable, and freely accessible atlas introduced in Frontiers in Plant Science. The magazine The Scientist has written about it, likening PubPlant to a kind of Google Maps for plant DNA, designed to help researchers more quickly pinpoint key genomic regions linked to traits such as disease resistance, nutritional quality, or climate adaptation. (Image Credit: Salk Institute-USDA)

Baby KJ in Nature’s Top Ten

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Every December, Nature selects the 10 people of the year, those who have most shaped the year that is coming to an end. For 2025, the little KJ Muldoon, about whom we have written many times, could not be left out. The first newborn to receive a CRISPR treatment developed specifically for him, the inspiration for new rules on the testing of advanced therapies for rare diseases, the mascot that patients, families, doctors, and scientists needed to look to the future of medical editing with renewed confidence.

Playing with Chromosomes: CRISPR’s New Frontier

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Humans have 23 pairs of chromosomes. The record-holder among animals (a butterfly called Polyommatus atlantica) can boast 229. Some plants have even more, but only because their genomes have undergone multiple rounds of duplication. We’re talking about chromosomes, of course. Their number is characteristic of each species and still shrouded in mystery. Why that number? And what would happen if we changed it?
In animals, the effects tend to be detrimental: mice with fused chromosomes, for instance, show abnormalities in behavior, growth, and fertility. Plants, however, appear surprisingly flexible, as demonstrated by a new experiment using CRISPR, recently published in Science.

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Can CRISPR resurrect Neanderthals?

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After the uproar over the de-extinction of dire wolves, bioethicist Arthur Caplan asked in Plos Biology: “Should scientists be allowed to bring distant human ancestors back to life?” The Italian edition of Scientific American invited me to investigate how technically difficult it would be to de-extinct a Neanderthal and what the risks and benefits of such a project might be. Below are the statements provided by the specialists I consulted.

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The FDA charts a new route for bespoke therapies

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Inspired by the Baby KJ case, the agency proposes a flexible framework allowing personalized treatments for individual patients to contribute to shared, platform-based approvals.

The announcement appeared on November 12 in the New England Journal of Medicine under a seemingly cautious title: “The FDA’s New Plausible Pathway.” Yet the article, written by two senior figures at the Food and Drug Administration, reveals vision and leadership. For once, it is worth starting from the end, which reads like a strong statement of intent: “Nearly 30 years after the sequencing of the human genome, bespoke therapies are close to reality. The FDA will work as a partner and guide in ushering these therapies to market, and our regulatory strategies will evolve to match the pace of scientific advances.”

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