Gene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments has been challenging.

Baby KJ Muldoon, the first patient to successfully receive personalized CRISPR gene editing therapy has returned home after over 300 days at the hospital.

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

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Last month, a baby got the world’s first personalized gene-editing treatment. What will this mean for millions of others with ...

Personalized CRISPR treatment used on baby with genetic disease by Steph Whiteside - 05/15/25 4:15 PM ET. by Steph Whiteside - 05/15/25 4:15 PM ET.

Bring up germline editing, and most scientists cringe. The idea behind the notorious CRISPR-baby scandal, editing reproductive cells or embryos tinkers with DNA far beyond just the patient—any changes ...

CRISPR, the gene-editing technique that can alter any cell and life-form on the planet, exemplifies the point. It can be deployed to alter a bird flu virus to kill multitudes. It can be used for ...

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

National Human Genome Research Institute, accessed Feb. 19, CRISPR Thank you for supporting our journalism. You can subscribe to our print edition, ad-free app or e-newspaper here .

In the latest case, the scientists developed a CRISPR treatment for a boy named KJ, who was born with genetic mutations in his liver cells that prevent him from breaking down proteins properly.

Babies born with CPS1 deficiency can receive liver transplants, but not until they are a year old, long enough that many have already suffered irreversible brain damage. Getting treatment to KJ ...