CRISPR technology is transforming modern medicine by enabling precise DNA editing to treat genetic disorders, including sickle cell anemia and beta-thalassemia, experts say.

A newly found organelle, the hemifusome, helps manage cellular recycling. It may play a role in inherited diseases and open ...

Gabriella Ngang, of Farmington Hills, is one of three in Michigan to take part in a clinical trial of a new sickle cell ...

This shift follows the announcement of initial positive data from the Phase 1/2 clinical trial of PM359 in Chronic ...

A remote Brazilian town's mystery illness was traced by biologist Silvana Santos to a rare genetic disorder, Spoan syndrome, ...

I have a 50 percent chance of inheriting the gene that causes the disease that killed my father. “I’ve decided to get tested ...

Psoriasis is a chronic condition where the body’s immune system causes skin cells to grow too quickly, leading to patches of ...

In a breakthrough study, scientists found that silencing a single overactive enzyme in the brain reversed early damage caused ...

Metabolic dysfunction-associated steatotic liver disease (MASLD) affects nearly one in three adults around the globe. It ...

One-time surgical application of genetically engineered autologous keratinocyte sheets provides significant closure and pain ...

Patients with rare diseases are increasingly working to find and fund their own cures. But should they have to?