Personalized Gene Editing

 Zaneyah Hughes

November 26, 2025

Genetics

Dr. Barbato

Personalized Gene Editing

    The Children’s Hospital of Philadelphia (CHOP) made history by being the first in the world to personalize the CRISPR gene-editing technology in order to save an infant’s life. The infant, named KJ, had a rare metabolic disease called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. Thanks to Ahrens-Nicklas and Kiran Musunuru, MD, PhD.'s years of preclinical research studying similar variants to KJ’s disease, they were able to target KJ’s variant soon after birth. 

    Not only is this use of CRISPR technology groundbreaking, but as it proved in KJ’s case, it's also life-changing. If KJ hadn’t have been treated with the gene editing therapy by the CHOP and Penn Medicine team, he would’ve been stuck with the general solution for patients with CPS1 deficiency. The problem with that is that he would have required a liver transplant, which requires the patient to be medically stable and old enough for it. Sadly, KJ was neither before the gene-editing therapy. Even though the CHOP and Penn Medicine team personalized KJ’s gene-editing therapy doesn’t mean that this was a solution unique only to him. I feel like Dr. Ahrens-Nicklas and Musunuru’s technique to save KJ can be applied not just to other patients with similar diseases, but many of people who have genetic diseases.

Source: https://www.chop.edu/news/worlds-first-patient-treated-personalized-crispr-gene-editing-therapy-childrens-hospital. 

Extra Source: https://innovativegenomics.org/news/first-patient-treated-with-on-demand-crispr-therapy/.