“US Doctors Use CRISPR to Cure Rare Liver Disorder in 9-Month-Old Baby”
In a historic milestone for precision medicine, doctors in the United States have successfully used a customised CRISPR-based gene-editing therapy to treat a nine-month-old infant suffering from a rare and life-threatening liver disorder. Baby KJ, diagnosed with carbamoyl phosphate synthetase 1 (CPS1) deficiency, became the first person in the world to receive a personalised DNA correction specifically designed to address his unique genetic mutation. The breakthrough, achieved by a team at the Children’s Hospital of Philadelphia (CHOP) and Penn Medicine, marks a revolutionary step in the treatment of ultra-rare genetic diseases.
A nine-month-old baby, KJ Muldoon, suffering from a rare genetic liver disorder, became the first person in the world to receive a personalized CRISPR-based gene-editing therapy. Developed by researchers at the Children’s Hospital of Philadelphia (CHOP) and Penn Medicine, this pioneering treatment marks a milestone in precision medicine and offers new hope for patients with rare, untreatable genetic conditions.
| Summary/Static | Details |
| Why in the news? | “US Doctors Use CRISPR to Cure Rare Liver Disorder in 9-Month-Old Baby” |
| Patient | KJ Muldoon, 9-month-old infant |
| Disorder | CPS1 deficiency (urea cycle disorder) |
| Therapy Used Personalized | CRISPR-based base editing |
| Institutions Involved | CHOP and Penn Medicine |
| Outcome | Improved protein tolerance, reduced medication, no major side effects |
| Global Significance | Opens doors for precision medicine in rare diseases |
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