Accumulation of clots causes some of the most severe symptoms of sickle cell disease, including strokes, kidney failure, ...

Four-week-old Tommy Parry is one of only 23 in the world ever to be born with the mitochondrial gene, which impairs his ...

In a novel theory, scientists at Arizona State University's Biodesign Institute propose a unifying explanation for the ...

The hypothesis puts forward the idea that stress granules disrupt the cell transport system, which then modifies gene ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

An international research team has identified key genetic factors that confer protective effects against Alzheimer's disease (AD) through a comprehensive genetic analysis of East Asian populations, ...

UMD researchers have discovered key mechanisms in gene regulation that could improve the design of RNA-based medicines.

A 66-year-old man has become the fourth person in the world to receive a genetically edited pig kidney transplant, performed ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

In a shift away from GMOS, aka genetically modified organisms, important research is continuing in gene editing, sometimes ...

On Friday, doctors at Massachusetts General Hospital announced that a 66-year-old New Hampshire man had successfully received ...

Older women could be vulnerable to harmful inflammation from new gene therapies to treat incurable eye diseases, new research ...