The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current CF therapies.

For decades, the standard explanation for autoimmune diseases like rheumatoid arthritis has rested on two pillars: inherited ...

A groundbreaking study published in this week’s issue of PNAS by scientists from Israel and Ghana shows that an evolutionarily significant mutation in the human APOL1 gene arises not randomly but more ...

CFTR is a protein essential to wet surfaces throughout your body, like the airways of your lungs. People with cystic fibrosis have a gene mutation that causes problems with the protein. Some ...

New research suggests that autoimmune diseases may be driven by DNA mutations in immune cells that remove the natural brakes on the immune system. It reveals a previously hidden role for somatic ...