CRISPR

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Illustration of CRISPR epigenome editing tool removing red methyl tags from a holographic DNA model to activate fetal globin genes, with sickle cell blood cells normalizing, in a modern research lab.
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CRISPR-based epigenome editing switches genes on by removing methyl tags, without cutting DNA

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Researchers at UNSW Sydney and St. Jude Children’s Research Hospital report a CRISPR-derived “epigenome editing” approach that turns genes on by removing DNA methylation marks rather than cutting DNA. In cell-based experiments, they show that promoter methylation can directly—and reversibly—silence fetal globin genes, a finding they say helps settle a long-running debate about whether methylation is causal or merely correlated with gene shutdown. The work points to a potential path toward safer therapies for sickle cell disease by reactivating fetal hemoglobin without creating DNA breaks.

Nobel Prize-winning scientist Jennifer Doudna has cofounded Aurora Therapeutics, a startup focused on developing personalized gene-editing treatments for rare diseases. The company plans to leverage CRISPR technology and a new FDA regulatory pathway to bring these therapies to market. This initiative builds on recent successes in custom treatments that have saved lives.

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Der Gesundheitsökonom Martin Morgenstern erklärte in einem Interview, dass Geneditierung die medizinischen Behandlungen in den kommenden Jahrzehnten transformieren wird. Seiner Ansicht nach ermöglichen Technologien wie CRISPR das Verändern spezifischer Gene, um Erkrankungen wie hohen Cholesterinspiegel zu bekämpfen. Dieser Ansatz verspricht präziser als traditionelle Medikamente zu sein, birgt jedoch inhärente Risiken.

Conservationists are divided over a proposed moratorium on genetic modification of wildlife at an upcoming International Union for Conservation of Nature meeting. While some groups seek a pause to assess risks, others argue that technologies like CRISPR are essential for saving endangered species. The vote could influence funding and policies worldwide.

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