The mum and dad of a baby with a genetic disease so rare it has no name have vowed to keep fighting for him after the ...

Hereditary high cholesterol (FH) is the most common hereditary disease and, if left untreated, can lead to atherosclerosis, ...

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

Postdoctoral scholar Daniela Soto has earned a prestigious fellowship from HHMI with eight years of funding to study the ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches -- gene therapy ...

When we can play any part in supporting other families, it’s a tragic gift that allows us to continue to parent Havi.” For families like the Goldstein-Sacks, genetic screening became a pivotal part of ...

Given the rise in newborn screening for ALD since 2019, and the untapped potential of existing genetic research, the Halls ...

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, ...

The CDC reports about 100,000 people in the United States are affected by Sickle cell disease, and more than 90% are non-Black or African American. One local organization is aiming to shed light on ...

Two new compounds may be able to treat retinitis pigmentosa, a group of inherited eye diseases that cause blindness. The compounds were identified using a virtual screening approach.