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 ...

1 Department of Pulmonary and Critical Care Medicine, The Third Xiangya Hospital, Central South University, Changsha, China 2 Department of Blood Transfusion, The Third Xiangya Hospital, Central South ...

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 ...