The article highlights David Liu's groundbreaking contributions to gene-editing technologies, particularly base editing. It discusses the significant impact of a recent case involving a 5-month-old boy, KJ, who received CRISPR treatment for a fatal genetic disorder. KJ's therapy aimed to correct a life-threatening mutation and led to his recovery, marking a milestone in medical science. Liu, recognized for his transformative work, emphasizes the importance of robust technology and teamwork in achieving rapid medical breakthroughs, showcasing that scientific advancements can significantly improve human lives.
There's a lot of confidence in base editing technology based on the 17 previous base editing clinical trials and thousands of research publications, but it doesn't change the fact that you still realize the stakes are very high for this patient and their family.
This story is a powerful testament to the fact that the editing technology, the delivery technology, the manufacturing, the animal models, and the regulatory approval are all robust enough to make this huge team effort happen fast enough to save Baby KJ.
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