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 was born. He got out of hospital on Tuesday.

After spending over 300 days at CHOP, KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene editing therapy.

Conclusions: In conclusion, glutamine synthetase expression are increased in fibrolamellar hepatocellular carcinoma. This study provides crucial insights to suggest a potential prognostic role on this ...

KJ Muldoon became the first patient to undergo personalized CRISPR treatment, a therapy that found the one uniquely mutated gene out of 20,000 in his little body, and fixed it.

A CRISPR treatment seems to have been effective for a baby’s devastating disease, but it is not clear whether such bespoke therapies can be widely applied ...

For the first time, doctors have created a customized treatment using the revolutionary gene-editing technique known as CRISPR to treat a baby with a rare, life-threatening genetic disorder.

Doctors save baby's life with first-ever gene fix for deadly rare disease The rare genetic disease kills 50% of babies diagnosed with it by early infancy.

Base editors can correct disease-causing genetic variants. After a neonate had received a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a disease with an estimated 50% mortality ...

Thrombotic microangiopathy rarely accompanies anti-synthetase syndrome; only about six cases of anti-synthetase syndrome with thrombotic microangiopathy have been reported, of which two cases had no ...

Glutamine synthetase (GS) is a ubiquitous enzyme central to nitrogen metabolism, catalyzing the ATP-dependent formation of glutamine from glutamate and ammonia. Positioned at the intersection of ...

Asparagine synthetase drives glutamine utilization in this disease and it is upregulated in human and mouse tissues. Its inhibition retards disease progression and rescues metabolic derangement in PKD ...