In 2023, doctors Ahrens-Nicklas and Musunuru began developing personalised gene-editing therapies for urea cycle disorders. After identifying KJ’s CPS1 variant at birth, they created a targeted base ...

CAMP4 Therapeutics announced promising results from its SYNGAP1 and Urea Cycle Disorders (UCDs) programs at the 28th Annual Meeting of the American Society of Gene and Cell Therapy. In ...

NEW ORLEANS – Investigators developed a personalized base-editing therapy for an infant with a urea cycle disorder and treated him with the ... The patient developed disease symptoms within 48 hours ...

A baby in Pennsylvania has become the first recipient of an experimental genetic therapy that targets a rare urea cycle disorder. The treatment, which involves customizing the gene editing process ...

Turned out, KJ was born with carbamoyl phosphate synthetase 1 (CPS1), an inherited genetic disease known as a urea cycle disorder. The condition causes toxic levels of ammonia to build up in a ...

Your son is very sick,' " says Kyle Muldoon, KJ's father. Turned out, KJ was born with carbamoyl phosphate synthetase 1 (CPS1), an inherited genetic disease known as a urea cycle disorder. The ...

Scientists were able to create a bespoke treatment for KJ Muldoon’s rare genetic disorder within six months. It could be a ...

Fargo’s Aldevron helped create a custom gene-editing therapy that cured a baby of urea cycle disorder, a rare and potentially fatal condition. A 9-month-old baby is recovering from a rare ...

Ahrens-Nicklas and Musunuru decided to focus on urea cycle disorders. During the normal breakdown of proteins in the body, ammonia is naturally produced. Typically, our bodies know to convert the ...

CPS1 deficiency is one of a group of metabolic diseases, so-called urea cycle disorders, that CHOP physicians have focused on as potentially curable with CRISPR gene editing. Dr. Petros Giannikopoulos ...

The pair hoped to make progress against urea-cycle disorders – inherited metabolic problems, like KJ’s that leave young children vulnerable to bursts of damaging ammonia. “I have a whole ...