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In a groundbreaking medical achievement, nine-month-old KJ Muldoon became the first person to receive a personalized gene-editing therapy, effectively treating his rare and often fatal genetic disorder, carbamoyl phosphate synthetase 1 (CPS1) deficiency. This milestone was made possible through decades of publicly funded research, highlighting the critical role of government investment in advancing medical science.
CPS1 deficiency is a rare metabolic disorder that impairs the body's ability to eliminate ammonia, leading to toxic accumulation. Traditionally, treatment options are limited, often requiring liver transplants. KJ's condition was dire, but a team at the Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania developed a customized CRISPR-based therapy to correct the specific genetic mutation causing his illness. The therapy was administered in early 2025, and KJ has since shown remarkable improvement, with reduced medication needs and better tolerance to illness.
Dr. Kiran Musunuru, a gene editing specialist from the University of Pennsylvania who co-authored the study that was published in the New England Journal of Medicine, said, "“This is the first step towards the use of gene editing therapies to treat a wide variety of rare genetic disorders for which there are currently no definitive medical treatments.”
This medical breakthrough is rooted in years of federally funded research. The National Institutes of Health (NIH) has been instrumental in supporting studies that led to the development of CRISPR technology. Notably, NIH funding for CRISPR-related research grew from more than $5 million in FY2011 to $1.1 billion in FY2018 . Such investments have been crucial in understanding and harnessing gene-editing technologies for therapeutic purposes.
In 2023, the NIH awarded a $14 million grant to researchers at Penn and CHOP to develop personalized gene-editing therapies for rare metabolic diseases using advanced CRISPR technology . This funding facilitated the rapid development of KJ's treatment, demonstrating how sustained public investment can lead to swift and life-saving medical innovations.
The success of KJ's therapy also underscores the importance of collaboration between public institutions and private entities. Looking ahead, experts believe that personalized gene-editing therapies could be adapted to treat a wide range of rare genetic diseases, many of which currently lack effective treatments. Dr. Rebecca Ahrens-Nicklas, part of the treatment team, expressed hope that KJ’s success will lead to more applications of personalized gene therapies.
