Treated with an individualized gene-editing therapy that corrects mutations directly on the genome, a patient shows ...

The baby boy with a rare urea cycle disorder received the bespoke therapy within a few months of birth and is doing well, ...

The baby boy received the bespoke therapy within a few months of birth and is doing well, according to a presentation at ASGCT.

PTC518’s mechanism of action involves promoting the inclusion of a pseudoexon in HTT mRNA, leading to its degradation and reduced ... Currently, there is no cure or therapy to delay or slow disease ...

Background: LRFN4, characterized by leucine-rich repeats and fibronectin type III domains, has been implicated in various human diseases. However, its role in immune regulation and cancer prognosis ...

Vaccines using mRNA are currently being studied for a wide range of diseases, including cancer, cardiovascular disease, ...

"We are unique in our ability to deliver this innovative treatment in such a short timeline," said Mark Wetzel, VP/GM mRNA CDMO Services at Aldevron. "This CRISPR therapy was made under ...

An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care ...

"We are unique in our ability to deliver this innovative treatment in such a short timeline," said Mark Wetzel, VP/GM mRNA CDMO Services at Aldevron. "This CRISPR therapy was made under exceptional ...