Diseases & Research

Sickle Cell Disease

By Dr Graham Beards (Own work) [CC-BY-SA-3.0 (http://creativecommons.org/licenses/by-sa/3.0)], via Wikimedia Commons

Hutchinson Center scientists recently developed new technologies in researching hemoglobinopathies to cure sickle cell disease and other hemoglobin diseases, utilizing our breakthroughs in gene editing.

Click the tabs below to learn more about our research ยป

Fast Facts

  • Sickle cell anemia is a disease passed down through families. The red blood cells which are normally shaped like a disc take on a "sickle" or crescent shape. Red blood cells carry oxygen to the body.
  • The prevalence of people with who carry the gene is within the range of 5-7% in the world and the number of new cases of affected infants is estimated at 300,000 per year. 
  • Sickle cell anemia is inherited from both parents. If only one parent has a sickle cell gene, then their offspring will only have the sickle cell trait. People with sickle cell trait do not have the symptoms of sickle cell anemia.
  • Almost all people with sickle cell anemia have painful episodes called crises. These can last from hours to days. Crises can cause intense pain in the lower back, leg, joints, and chest.
  • In the past, people with sickle cell disease often died between ages 20 and 40. Thanks to a better care people now can live to the age of 50 and beyond. Causes of death include organ failure, cardiac and pulmonary complications, liver failure, stroke and infections.

[back to top]

Treatment & Prognosis

Increasing levels of fetal hemoglobin - Scientists have known for 50 years that increase levels of fetal hemoglobin (HbF) can cure sickle cell disease, yet extensive research has not yielded new viable HbF-inducing agents. Researchers at the Hutchinson Center have recently defined Bcl11a as key silencer of HbF, thus generating a new paradigm for treatment: inhibiting Bcl11a function. Recapitulating a naturally occurring mutation in humans who inhibit Bcl11a's function can lead to therapeutic levels of fetal hemoglobin in sickle cell patients.

Correcting genes that cause inherited diseasesDr. Barry Stoddard and his team of researchers made a leap forward when they determined the three-dimensional structure of a protein called a TAL effector – transcription-activator-like effector – which, along with a group of DNA-targeting enzymes called homing endonucleases, can be used to treat genetic diseases. Dr. M.A. Bender identified a DNA sequence associated with sickle cell disease.  Stoddard and team have engineered a targeting protein to moderate the genetic defect that causes sickle cell disease. Learn more »

[back to top]

Seattle Cancer Care Alliance logo

Fred Hutch's clinical research breakthroughs are delivered to patients through our treatment arm, the SCCA.

Make an appointment
Clinical trials unit patient lounge

Clinical trials are vital to the development of innovative treatments for Sickle.

Find a Clinical Trial
Researcher in lab

Fred Hutch scientists are producing some of the most important breakthroughs in the prevention, early detection and treatment of cancer, HIV and other diseases.

Donate to our Research
Fred Hutchinson Cancer Research Center is a world leader in research to prevent, detect and treat cancer and other life-threatening diseases.