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From birth to gene-edited in 6 months: Custom therapy breaks speed limits
The researchers described the unprecedented speed of the oversight steps as being "through alternative procedures." In month five, they started toxicology testing in mice. In the mice, the experimental therapy corrected KJ's mutation,
A Delaware County infant with a rare metabolic disease, CPS1 deficiency, was treated at CHOP with a custom CRISPR therapy.
News Medical on MSN4d
New gene editor evoCAST enables precise insertion of complete genesAsk scientists what gene editing tool is most needed to advance gene therapy, and they'd probably describe a system that's now close to realization in the labs of Samuel Sternberg at Columbia ...
To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of ...
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