UCLA researchers led by Dr. Donald Kohn have created a method for modifying blood stem cells to reverse the genetic mutation that causes a life-threatening autoimmune syndrome called IPEX. The gene therapy, which was tested in mice, is similar to the technique Kohn has used to cure patients with another immune disease, severe combined immune deficiency, or SCID, also known as bubble baby disease.
Strokes happen when an obstruction restricts the blood supply to the brain, and so the brain does not receive enough oxygen. A person's genetic makeup and their lifestyle both influence their risk of stroke, but which is more important?
A City of Hope (a leading research and treatment center for cancer, diabetes and other life-threatening diseases) scientist has discovered a gene-editing technology that could efficiently and accurately correct the genetic defects that underlie certain diseases, positioning the new tool as the basis for the next generation of genetic therapies.
Like a genetic handyman, an elusive enzyme deep inside certain cells repairs the tips of chromosomes, which fray as cells divide. It’s prized by rapidly dividing cells – like stem cells and tumor cells – and by scientists on the hunt for cancer and other disease therapies.
Researchers from the University of Liverpool identified a genetic variant that could improve the safety and effectiveness of corticosteroids, drugs that are used to treat a range of common and rare conditions including asthma, and chronic obstructive pulmonary disease (COPD).