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Showing posts with the label gene-editing

Baby with rare disease given world-first personal CRISPR gene therapy

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An infant with a severe genetic condition has shown signs of improvement after receiving a gene-editing treatment tailored to his specific mutation Originally published   by Michael Le Page at newscientist.com on 15 May 2025 Baby KJ after a gene-editing infusion with researchers Rebecca Ahrens-Nicklas and Kiran Musunuru Children’s Hospital of Philadelphia A baby boy with a life-threatening genetic condition has become the first person to receive a bespoke CRISPR gene-editing treatment , giving a glimpse into what t he future of medicine might look like. It’s the first time anyone has been given a gene-editing treatment designed t o correct a disease-causing mutation found only in that individual, Rebecca Ahrens-Nicklas at the Children’s Hospital of Philadelphia, Pennsylvania , told a press briefing. “He’s showing some early signs of benefit,” she says, but it is too soon to tell how well the treatment worked. The researchers published the details as soon as possible...

New CRISPR tool accelerates and optimizes genome editing

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Originally published by April Wendling, University of Illinois at Urbana-Champaign, on February 13, 2024 CRISPR-COPIES has applications in synthetic biology toolkit characterization, gene therapy, and metabolic engineering. Credit: Aashutosh Boob et al. CRISPR/Cas systems have undergone tremendous advancement in the past decade. These precise genome editing tools have applications ranging from transgenic crop development to gene therapy and beyond. And with their recent development of CRISPR-COPIES , researchers at the Center for Advanced Bioenergy and Bioproducts Innovation (CABBI) are further improving CRISPR's versatility and ease of use . "CRISPR-COPIES is a tool that can quickly identify appropriate chromosomal integration sites for genetic engineering in any organism ," said Huimin Zhao, CABBI Conversion Theme Leader and Steven L. Miller Chair of Chemical and Biomolecular Engineering (ChBE) at the University of Illinois . "It will accelerate ...