UCSF scientists have developed a new strategy to precisely modify human T cells using the CRISPR/Cas9 system.
Mongersen: A hope for patients suffering for Crohn’s disease
Crohn’s disease is a relapsing inflammatory bowel disease, which is mainly associated with severe inflammation of intestinal and colonic mucosa in response to a dysregulated immune system. The usual clinical symptoms of Crohn’s disease are abdominal pain, fever, and bowel obstruction or diarrhea with passage of blood or mucus. The diagnosis of the disease is largely based on the clinical symptoms, histology, laboratory parameters, and endoscopic examination to evaluate the changes taking place in the gastrointestinal tract. The exact etiology of the disease is not known yet, but a number of factors, such as genetic influences and malfunctioning of immune system, most likely to play a pivotal role in the development of the disease. Crohn’s disease is characterized by reduced activity of the immunosuppressive cytokine transforming growth factor β1 (TGF-β1), because of high levels of SMAD7, an inhibitor of TGF-β1 signaling.