Alpha-1 antitrypsin deficiency (AATM) is a congenital metabolic disorder. It is caused by a genetic defect, which results in ...

Genetic analysis of nearly 50,000 people identified carriers of sickle cell and beta-thalassemia traits, many of whom lacked ...

Setara Biosciences, Inc. (Setara), a clinical-stage biotechnology company leveraging genetics to develop precision medicines ...

Single-cell profiling of nearly 2,000 people brings scientists closer to understanding the link between genetic variation, gene expression and health.

Motor neuron diseases, such as amyotrophic lateral sclerosis (ALS) and hereditary spastic paraplegia (HSP), share physical similarities but have been largely viewed as genetically distinct. However, ...

IgA vasculitis is a pediatric disease with skin and systemic manifestations. Here, the authors use multiomic data to examine the genetic mechanisms of IgA vasculitis and find therapeutically relevant ...

Whole-genome embryo screening is expanding what prospective parents can learn during IVF. Instead of checking only chromosome ...

Gene therapy is no longer the future of medicine—it's reality. Treatments that replace, silence or edit genes are already ...

In three studies published in Nature today, Australian researchers have made advances in our understanding of how genetic ...

There are some 80 human diseases that in one way or another are considered autoimmune. Perhaps the most common indication of an autoimmune pathogenesis is the presence of circulating antibodies that ...

Ohio's six-month freeze on new Medicaid home care and hospice providers is set to expire Nov. 14, raising concerns among disabled residents who face gaps when aides leave. South Dakota voters will ...

Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs). The Company is ...