Doctors edit infant’s DNA in historic treatment for rare disorder
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Doctors in the U.S. have successfully used a customized gene-editing therapy to treat an infant with a life-threatening genetic disorder, marking a major milestone in medicine. The condition, which often proves fatal in early infancy, was identified shortly after birth. Specialists at the Children’s Hospital of Philadelphia and the University of Pennsylvania developed and tested the personalized treatment in just six months. Experts say the breakthrough highlights the potential for rapidly tailored therapies to correct genetic diseases early in life.
Source
Musunuru, K., Grandinette, S. A., Wang, X., Hudson, T. R., Briseno, K., Berry, A. M., Hacker, J. L., Hsu, A., Silverstein, R. A., Hille, L. T., Ogul, A. N., Robinson-Garvin, N. A., Small, J. C., McCague, S., Burke, S. M., Wright, C. M., Bick, S., Indurthi, V., Sharma, S., Jepperson, M., … Ahrens-Nicklas, R. C. (2025). Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. The New England journal of medicine, 392(22), 2235–2243. https://doi.org/10.1056/NEJMoa2504747
Additional Reading
https://abcnews.go.com/Health/doctors-save-babys-life-gene-fix-deadly-rare/story?id=121810273