CRISPR is a promising treatment for blood disorders, but it comes with challenges, such as off-target effects.

Cleveland Clinic researchers depleted BACE1 to reverse amyloid plaque formation in mice with Alzheimer's disease.

A stem cell pathway could be targeted to create more effective treatments for triple-negative breast cancer.

A novel therapy enhances the reception of the cartilage growth signals while blocking the inflammatory ones that lead to joint degeneration.

French scientists discovered that an X chromosome-linked abundance of a toll-like receptor could be the root cause of lupus.

European researchers discovered how drugs bind to the enzyme DHODH, which could lead to more selective cancer drugs.

A team at Duke University has created the first functioning human muscle from skin cell-derived induced pluripotent stem cells.

Alnylam, which put its hemophilia program on hold in September, has gotten the all-clear from the FDA to resume trials.