CRISPR Gene Therapy Saves Infant With Rare Genetic Disorder

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GenEdited Baby

CRISPR-based therapy successfully treated an infant with a rare genetic condition. Marking the first successful individual CRISPR treatment.

• CRISPR = Clustered regularly interspaced short palindromic repeats

• Condition: CPS1 deficiency, prevents nitrogen breakdown

• Treatment developed in just 6 months

Treatment was delivered to the liver via lipid nanoparticles and the infant quickly showed improved weight gain and reduced medical needs. A breakthrough that paves the way for future personalized CRISPR therapies.

Fun fact: I wrote this from a cafe in a CRISPR office!

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