Baby KJ Muldoon Gene Editing Success

K. J. Muldoon, a baby born with severe CPS1 deficiency, has successfully received an experimental personalized gene editing treatment. Developed by Children s Hospital of Philadelphia and University of Pennsylvania Health System, the therapy used a 'base editing' technique of CRISPR to correct a genetic error. This case, described in the The New England Journal of Medicine by researchers including Kiran Musunuru and Rebecca Ahrens-Nicklas, marks a significant step towards treating a wide variety of rare genetic disorders. The treatment, partially funded by the United States — National Institutes of Health, has allowed K. J. Muldoon to thrive, with researchers hoping to apply lessons learned to other rare disease patients. Experts like Senthil Bhoopalan and Carlos Moraes view this as a benchmark-setting advance that will accelerate the entire field of gene therapy.