RNA therapeutics target translation rather than DNA, aiming to correct shared protein production errors. By enabling cells to read through premature stop codons, engineered tRNAs could restore ...

Early embryonic arrest (EEA) can result in repeated failures of assisted reproductive technology, with genetic variation being the primary cause. The maternal protein nucleotide-binding ...

NEW YORK – PTC Therapeutics has withdrawn a new drug application (NDA) for its Duchenne muscular dystrophy drug Translarna (ataluren) that it had submitted for US Food and Drug Administration review.

Researchers at the Broad Institute, Harvard University, and the University of Minnesota have come up with a new gene-editing method, called PERT, that could one day treat a bunch of rare genetic ...

In all patients, results of a platelet factor 4 (PF4)–induced platelet-activation (PIPA) assay were strongly positive, and serum from all the patients tested strongly positive against PF4 alone in a ...