Wednesday, December 07, 2011

Salk researchers develop safe way to repair sickle cell disease genes New gene editing technique would heal patients with their own cells

Source: Salk Institute for Biological Studies
Date: December 7, 2011

Summary:

LA JOLLA, CA—Researchers at the Salk Institute for Biological Studies have developed a way to use patients' own cells to potentially cure sickle cell disease and many other disorders caused by mutations in a gene that helps produce blood hemoglobin. The technique uses cells from a patient's skin to generate induced pluripotent stem cells (iPSCs), which are capable of developing into various types of mature tissues—including blood. The scientists say their method, which repairs the beta-globin gene (HBB), avoids gene therapy techniques that can introduce potentially harmful genes into cells. The new technique, which will soon be tested as a therapy in animals, also appears to be much more efficient than other methods tested to date, the researchers say. The study appears in the December 2011 issue of Cell Research.