Using CRISPR, Hebrew University of Jerusalem team finds PEDS1 enzyme linked to reduced brain size; study maps 331 genes ...

There are currently no approved disease-modifying therapies for MPS IIIA.

OGM was selected as a genome-wide, unbiased method to detect large genomic rearrangements and structural variants (SVs) with sensitivity to variant allele fractions (VAFs) as low as 5% to analyze ...

If regionalization is being seriously considered, residents deserve clear answers to basic questions that remain unanswered.

Plants make chemical weapons to protect themselves, and many of these compounds have become vital to human medicine. Researchers found that one powerful plant chemical is produced using a gene that ...

Skeletal muscle and cardiovascular diseases continue to pose significant challenges to public health, affecting a wide range ...

Scientists at The University of Texas at Austin have developed a revolutionary gene-editing method using bacterial retrons that can correct multiple disease-causing mutations at once. Unlike ...

The tool helps scientists understand how single-letter mutations and distant DNA regions influence gene activity, shaping ...

An epidemiology study finds variations in one gene, APOE, play a major role in determining the risk of Alzheimer’s disease.

This year marked a pivotal moment in the quest to treat Huntington’s disease, a rare but devastating form of dementia. Scientists found that an experimental gene therapy slowed the condition’s ...

KJ Muldoon received a groundbreaking new treatment earlier this year. The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...