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

Baby KJ Muldoon was born with a rare genetic disorder called CPS1 deficiency and spent nearly the entire first year of his ...

KJ Muldoon's life-threatening disorder, called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, was treated with ...

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

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

University of Haifa study shows how genetic "imprinting" determines whether rare syndrome leads to autism or psychosis, offering new model for personalized psychiatry.

The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare ...

The child will need to be monitored throughout his life, but researchers said the treatment’s success indicates that other ...

The one-off treatment highlights the promise of a new type of gene editing and the challenges of using it to treat extremely rare genetic diseases.

Mansour is set to make medical history as the first person in the world to receive treatment for NARS1, a rare genetic disorder. (Juan Figueroa / Staff Photographer) An 11-year-old from Rowlett is ...

PURA, and CTNNB1—in human cell cultures and animal models. We stand on the brink of medical breakthroughs—we are so close. This research addresses a fundamental challenge in treating genetic disorders ...

PURA, and CTNNB1—in human cell cultures and animal models. We stand on the brink of medical breakthroughs—we are so close. This research addresses a fundamental challenge in treating genetic disorders ...