Rare genetic diseases are challenging for patients and their families—made all the more overwhelming because symptoms tend to appear soon after birth. To date, there haven’t been many reliable ...

A baby boy with a devastating genetic disease is thriving after becoming the first known person to receive a bespoke, CRISPR therapy-for-one, designed to correct his specific disease-causing mutation.

Angiopoietin-like protein 3 (ANGPTL3) inhibits lipoprotein and endothelial lipases. ANGPTL3 loss-of-function genetic variants are associated with decreased levels of low-density lipoprotein ...

Jon LaPook, M.D. is the award-winning chief medical correspondent for CBS News. Since joining CBS News in 2006, LaPook has delivered more than 1,200 reports on a wide variety of breaking news and ...

Cutting-edge gene editing technology could eradicate Down syndrome, according to Japanese scientists. Down syndrome, which causes a range of developmental differences and affects 1 in 700 newborns in ...

The world's first patient to be treated with personalized gene editing therapy is finally headed home after over 300 days in the hospital. Nine-month-old KJ Muldoon recently underwent CRISPR-based ...

In the first 48 h after K.J. Muldoon’s birth last year, doctors noticed signs of lethargy and respiratory distress. A blood test revealed off-the-charts ammonia levels—they exceeded 1,000 µmol/L, ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...

Dr. Musunuru and Dr. Ahrens-Nicklas holding KJ post infusion Senior Correspondent Gene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments ...