New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

No gene acts alone: interacting variants and protein partnerships can worsen, mask or even rescue disease risk, demanding ...

The FDA granted fast track status to VectorY’s VTx-002, an antibody gene therapy targeting TDP-43 protein aggregates in ALS.

Precision and timing of gene expression is essential for normal biological functions and, when disrupted, can lead to many ...

More than 9 out of 10 Alzheimer's cases could be driven by specific variations in a single gene and the protein it produces, ...

Non-protein-coding genes have been linked to a hereditary condition, retinitis pigmentosa, that causes progressive blindness.

An international research team has succeeded in deciphering a key mechanism that controls the growth of pancreatic cancers. The scientists identified a potential central mechanism by which cancer ...

In a groundbreaking study published in the in Proceedings of the National Academy of Sciences (PNAS), scientists at Pacific ...

BUFFALO, N.Y., — Many childhood cancers start with a hijacking at the molecular level. A group of abnormal proteins known as fusion proteins aberrantly engage with a collection of proteins that ...

The functioning of neurons, cells that transmit information in the nervous system, heavily relies on the production of ...

Many childhood cancers start with a hijacking at the molecular level. A group of abnormal proteins known as fusion proteins aberrantly engage with a collection of proteins that switches genes on and ...