Despite widespread perceptions that 2025 was a dismal year, significant advancements were made, including a breakthrough in genetic medicine. A notable story involves the birth of KJ Muldoon, who arrived with an extremely rare genetic disorder known as carbamoyl phosphate synthetase 1 deficiency, which can be lethal.
In an impressive collaboration, the Children’s Hospital of Philadelphia and the University of California-Berkeley developed a personalized CRISPR-based therapy to address KJ’s condition. This innovative approach allowed for the correction of a single genetic mutation, which was critical for KJ’s survival.
In February, the FDA granted emergency authorization for the treatment, marking a significant milestone in gene-editing therapies aimed at serious genetic disorders. While many discussions around 2025 center on its challenges, such as political instability and societal issues, the success in genetic research provides a hopeful narrative amid the turmoil.