Multisystemic smooth muscle dysfunction syndrome (MSMDS) is a rare condition associated with stroke, aortic dissection ...

In a major step toward treating rare neurological diseases, scientists have used gene editing to correct mutations inside the brain of living mice with a single injection. This new approach, described ...

CRISPR gene-edited cell therapy improves quality of life in patients with transfusion-dependent beta-thalassemia, research suggests.

Researchers at Mass General Brigham in Boston have designed a custom gene editing technique that corrected the mutation ...

Editing genes involves changing the sequence of letters in the DNA. Researchers like to edit genes so they can understand the function of them, particularly genes that relate to various types of ...

The commercial success of existing lipid-lowering medications highlights the enormous market potential for effective ...

Gene therapy has long promised to provide a solution for genetic blood disorders, but delivering on that promise has proven complex due to challenges including difficulties delivering the editing ...

Multisystemic smooth muscle dysfunction syndrome (MSMDS) is a rare condition associated with stroke, aortic dissection (tearing) and death in ...

Vitamin D plays a vital role in the human body, far beyond just keeping bones strong. It helps your muscles move, allows your ...

During the Neogene period, our ancestors produced a useful enzyme that lowered uric acid levels. Gene-editing is reviving ...

Researchers used evolutionary genetics and CRISPR gene editing tech to develop an innovative treatment for gout. The approach ...