Examples of human genome editing research

Research is currently underway on clinical applications of genome editing technologies to treat the following genetic disorders:

  • sickle cell disease (red blood cells taking a crescent shape and getting stuck in small blood vessels leading to organ damage, especially in the lungs, kidneys, spleen, and brain),
  • cystic fibrosis (the production of thick and sticky mucus, sweat or digestive juices which damages the lungs, digestive system and other organs),
  • congenital (present at birth) blindness usually caused by inherited eye diseases,
  • haemophilia (inherited bleeding disorder where the blood does not clot properly),
  • amyloidosis (abnormal proteins build up in organs, such as the heart, kidneys, liver, and can lead to their failure), and
  • lysosomal storage disorders (abnormal build-up of toxic materials in cells as a result of enzyme deficiencies affecting e.g. the skeleton, brain, skin, heart, and central nervous system).

In addition, significant progress in therapeutic genome editing has been demonstrated in cancer and infectious diseases, such as HIV and Hepatitis B.