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World-first CRISPR therapy
June 3 , 2025
4 days
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- The baby, KJ Muldoon from Pennsylvania of USA, is one of 350 million people worldwide with a rare disease - CPS1 deficiency.
- This baby became the first to successfully receive personalized gene therapy.
- The Doctors' team created a therapy designed to correct KJ’s faulty gene.
- They used CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020.
- Doctors employed a technique known as “base editing.”
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