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#CRISPR isn’t living up to its potential. Thousands of rare diseases that could be treated with gene editing remain untouched by drug companies. Two scientists, Fyodor Urnov + Kiran Musunuru, have a plan to fix that. Read more in my deep dive for Endpoints News: endpts.com/crispr-isnt-li…

CHOP & Penn Medicine have delivered the first-ever personalized gene editing therapy for a patient with CPS1 deficiency, marking a major milestone in the application of CRISPR-based treatments. Learn more about KJ & the future of personalized medicine: ms.spr.ly/6015SZLAx.

BREAKING: Last August, scientists secretly began creating a custom #CRISPR therapy to fix a mutation responsible for one baby’s debilitating disease. He was treated in February and doing better. My deep dive in Endpoints News has the details. endpts.com/philadelphia-d…

Breaking News: A baby with a rare disorder made medical history by receiving the first custom gene-editing treatment. The technique used has the potential to help people with thousands of other uncommon genetic diseases. nyti.ms/4j3xGs8

So happy to have helped make this personalized gene editing therapy a reality for KJ! Hopefully, this workflow can be applied to more N-of-1 patient variants to treat severe genetic diseases.

A few weeks ago we introduced you to KJ, who made medical history as the first patient to receive a personalized gene editing therapy. After almost a year at CHOP, KJ is now at home with his family. 🎓 Tune in to NBC's TODAY tomorrow morning to hear from his family!

.Secretary Kennedy - thank you so much for your broad support for gene editing and gene therapies as well as recognizing the importance of Baby KJ’s story and the potential that CRISPR base editing therapies hold for patients with severe genetic diseases. I’m thrilled you have publicly