New gene therapy improves hearing in patients with rare genetic deafness
A new international study co-led by investigators from Mass General Brigham and the Eye & ENT Hospital of…
Antisense oligonucleotides reverse rare neurodevelopmental disorder in preclinical models
Scientists at St. Jude Children’s Research Hospital have found they can reverse the effects of HNRNPH2-related neurodevelopmental disorder using…
Early genomic testing prevents years of inconclusive visits for pediatric patients
For many families, eight years of uncertainty meant repeated specialist visits, inconclusive genetic tests and the growing fear…
APC-deficient cancer cells rely on single enzyme for survival
A newly published study reports that APC-deficient cancer cells may depend on a single metabolic enzyme for survival,…
New gene target offers hope for fatal infant cardiomyopathy
Researchers from the Keck School of Medicine of USC have made an important advance toward understanding-and potentially treating-a rare cardiomyopathy…
Gut microbiome analysis may help detect Parkinson’s before symptoms appear
Analysis of microbes in the gut can reveal whether a person faces an elevated risk of Parkinson’s disease,…
Proteomics and AI bring earlier risk prediction into sharper focus
From Alzheimer’s risk to cardiovascular prediction, this review shows how protein profiling and AI could help detect disease…
Key gene variants tied to developmental dysplasia of the hip and osteoarthritis
Developmental dysplasia of the hip (DDH) is a polygenetic disorder that increases the risk of hip osteoarthritis. A…
How the immune system may influence Alzheimer’s, Parkinson’s, and related diseases
A sweeping review shows how the brain’s immune defenses can both fight and fuel neurodegeneration, revealing why timing,…
Epigenetic changes allow cancer cells to shift identity and survive drugs
Cancer drugs are designed to shut tumors down. But sometimes, in the very act of attacking a tumor,…
Migraine genes linked to worse headaches after concussion in children
A University of Calgary-led study has found evidence that children with genes predisposing to migraine might have an…
Study identifies new genes linked to severe pregnancy sickness
The USC research team that recently identified the hormone-encoding gene GDF15 as a key driver of pregnancy sickness has identified…