We recently reported evidence that FAD mutations in APP and PSEN1 cause deficiencies in early steps in processive proteolysis of APP substrate C99 and that this results from stalled γ-secretase enzyme ...

CRISPR gene editing based treatment reduced HAE attacks rates by up to 80%. NTLA-2002 was well tolerated by patients. The ...

OHSU is one of 20 sites hosting the clinical trial for the amyloidosis therapy. Masri, who is overseeing the trial in Oregon, told The Lund Report that finding a way to deliver gene therapy directly ...

Researchers have used whole-genome CRISPR interference screening to identify the “Achilles Heel” of drug-resistant ...

Gliocidin selectively kills glioblastoma cells by inhibiting nucleotide synthesis, showing potential as a therapeutic option ...

By targeting the TTR gene directly in the liver, the therapy “has opened up the door” to permanent treatment, says Sarah ...