Infant with Rare, Fatal Genetic Disorder Becomes First to Receive Personalized CRISPR-Based Gene Therapy
In a historic first for precision medicine, a research team supported by the National Institutes of Health (NIH) has successfully delivered a personalized CRISPR-based gene-editing therapy to treat an infant diagnosed with a rare and life-threatening genetic condition—carbamoyl phosphate synthetase 1 (CPS1) deficiency. The groundbreaking treatment marks the first known use of patient-specific CRISPR gene […]
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