Baby Receives World's First Personalized Gene Therapy, Shows Positive Recovery

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A nine-and-a-half-month-old baby, KJ Muldoon, from Clifton Heights, Pennsylvania, has become the first patient globally to receive a bespoke gene therapy specifically designed for his rare metabolic condition, severe carbamoyl phosphate synthetase 1 deficiency. After spending months in a hospital on a restrictive diet, KJ was administered the groundbreaking treatment in February, followed by doses in March and April.

According to KJ’s father, Kyle Muldoon, the family extensively researched options before opting for this experimental treatment. Since then, KJ has shown remarkable improvement, allowing him to eat more normally and recover from common illnesses.

Experts, including Dr. Rebecca Ahrens-Nicklas from the Children's Hospital of Philadelphia, emphasized that KJ's case represents a critical step towards using gene editing therapies for various rare genetic disorders. Utilizing the CRISPR gene-editing tool, researchers replaced a mutation in KJ’s DNA responsible for his condition. Dr. Kiran Musunuru, a co-author of the study, noted that rapid publication of these results could pave the way for more personalized treatments in the future.

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