Genetic medicine has made huge progress over the past two decades. Gene therapy, gene editing, and mRNA treatments are now approved for a growing number of conditions. These approaches focus on fixing ...

The firm's first candidate, AP003, will focus on rare genetic liver diseases caused by nonsense mutations that prematurely halt protein production.

tRNA is an important component of the biological synthesis of new proteins, as it carries a specific amino acid that will be incorporated to the protein through the ribosome machinery, as directed by ...

A groundbreaking study has shed light on the critical role of a tRNA methylation enzyme, TRMT10A, in supporting brain function. The findings reveal how the absence of TRMT10A leads to a reduction in ...