One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...

The Rice lab of bioengineer Gang Bao and collaborators at Baylor College of Medicine have developed a new gene-editing strategy that dramatically boosts the effectiveness of gene therapies in the ...

Researchers at Washington University School of Medicine in St. Louis have conducted a longitudinal study on an individual ...

It is an especially relevant question for many living with Huntington’s Disease (HD). Notably, some studies have shown that ...

Your genes play a huge role in what makes and shapes your entire existence. Since you inherit your genes from your parents, ...

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

The manuscript addresses the 3D chromatin architecture in monocytes from patients with alcohol-associated hepatitis and its relationship to enhanced transcription of innate immune genes. While the ...

Families of rare disease patients appeal to PM Modi and Health Minister for increased treatment funding support.

Doug Whitney was supposed to develop Alzheimer's by 50. Now scientists are trying to understand why his brain remains healthy ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or inactivating genes. Precisely fixing disease-causing mutations is far more ...

By Julie Parr Click here for updates on this story TEXARKANA, Texas (KTBS) — A Texarkana, Texas boy battling a rare genetic ...

WORCESTER ― A foundation that honors the life of a Massachusetts man who grew his family's produce business from a pushcart ...