A BABY RECEIVED A CUSTOM CRISPR TREATMENT IN RECORD TIME
https://media.wired.com/photos/6824ccff0345efd6df217889/master/pass/Infant%20KJ%20wrapped.JPG
✍️ A 6-month-old baby with a rare genetic disorder received a personalized Crispr treatment, showing promising results and potentially paving the way for customized gene-editing therapies for other patients.
👉 KJ Muldoon, a 6-month-old baby, was born with CPS1 deficiency, a rare genetic disorder that causes ammonia buildup in the blood. 👉 The disorder has no ideal treatment options, and about half of babies born with it will die early in life. 👉 A team at the Children's Hospital of Philadelphia and Penn Medicine designed a customized Crispr treatment for KJ, bypassing the standard years-long drug development timeline. 👉 The treatment has shown significant improvement in KJ's condition, allowing him to eat certain foods and sit upright. 👉 The case highlights the potential of Crispr technology to directly address the underlying genetic cause of diseases. 👉 The team's approach could serve as a blueprint for making customized gene-editing treatments for other patients with rare diseases.
-- #KiranMusunuru #RebeccaAhrens-Nicklas #TerryHorgan #TimothyYu #FyodorUrnov #KyleMuldoon #Children'sHospitalofPhiladelphia #PennMedicine #UniversityofPennsylvania #NewOrleans #BostonChildren'sHospital #UCBerkeley #health
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