Sickle Cell Disease

Diseases / Research

Sickle Cell Disease

sickle cell

This is a peripheral blood smear with many normal red blood cells that are round with clear centers. The abnormal sickle cells have change their morphology due a mutation in hemoglobin and low oxygen to a sickle shape that appear as a enlongated linear cell with a cigar shape. Because of the abnormal morphology these cells have problems passing through small capillaries blocking the flow of blood and causing ischemic crisis.

Photo by Keith Loeb, PhD, and Sindhu Cherian, MD, SCCA Department of Pathology

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

Hutch researchers identified the DNA sequence associated with sickle cell disease and are continuing their efforts to develope therapies for the disease.

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.

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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 >

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