Groundbreaking CRISPR Gene Editing Therapy Saves Baby KJ Muldoon
Baby KJ Muldoon, the first patient to successfully receive personalized CRISPR gene editing therapy, has returned home after over 300 days at the hospital. In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children’s Hospital of Philadelphia (CHOP) and Penn Medicine.
KJ Muldoon was born with a rare metabolic disease known as severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. After spending over 300 days at CHOP, KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene editing therapy.
The effort to treat KJ was led by Dr. Rebecca Ahrens-Nicklas, director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program at CHOP, and Dr. Kiran Musunuru, a renowned geneticist at Penn’s Perelman School of Medicine. For the first time, doctors have treated a baby born with a rare, life-threatening genetic disorder with a gene-editing therapy tailored to specifically repair his unique mutation.
KJ Muldoon’s successful treatment marks a significant milestone in the field of personalized medicine and gene editing therapies. This groundbreaking CRISPR therapy has given hope to patients with rare genetic disorders and paves the way for future advancements in medical science.
