Sometimes, in genetics, two wrongs do make a right. A research team has recently shown that two harmful genetic variants, ...

BIRMINGHAM, Ala. – Modifier genes add complexity to precision medicine. A modifier gene is one that has genetic variants such as single nucleotide changes that differ from the most common sequence in ...

Phase 2 (~50% of patients evaluated to date at 12 months) shows 46% lesion growth reduction vs. controlThere are no OCU410-related serious ...

Cystic fibrosis is caused by an inherited mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Due to this mutation, the CFTR protein doesn't embed in cell membranes to ...

Scientists have developed a new way to understand complex genetic diseases and have identified a gene that modifies the severity of inherited kidney disease, paving the way for personalised treatments ...

Sometimes genetic diseases are not as straightforward as they seem. Charles Homcy learned that firsthand more than four decades ago when he was a medical student at Johns Hopkins University. During ...

Sickle-cell disease is caused by a one-letter spelling mistake in the hemoglobin gene. But not everyone who inherits the error suffers the worst effects of the blood ...

Neurodevelopmental disorders, including schizophrenia and autism, likely result from complex interactions that modify the effects of individual genes, according to new research. Neurodevelopmental ...

Steven Pittler, Ph.D., at the University of Alabama at Birmingham has sought to find modifier genes for the hereditary eye disorder retinitis pigmentosa type 59. After onset in one’s late teens, RP59 ...