Mayo Clinic Minute: Sickle cell disease explained

The Food and Drug Administration is expected to decide by Dec. 8 on a new therapy to treat sickle cell disease using gene editing technology called CRISPR, which stands for clustered regularly interspaced short palindromic repeats.

Approximately 250 million people worldwide carry the gene for sickle cell disease. It affects those with roots in Africa, Spanish-speaking regions in the Western Hemisphere, Saudi Arabia, India and Mediterranean countries. In the U.S., sickle cell disease is most common in the African American community. 

Dr. Asmaa Ferdjallah (ESS-mah fer-JALL-ah) a pediatric hematology, oncology and bone marrow transplant physician, is the medical director of Mayo Clinic Children's Center's Comprehensive Pediatric Sickle Cell Program. She says the red blood cell disorder is a lifelong condition with unique challenges.

Mayo Clinic's Dr. Asmaa Ferdjallah (esmah fer-jallah) says the normally flexible red blood cells turn into rigid crescent shapes — sickle cells— that get stuck in blood vessels. And that can cause problems down the line.

Babies may not show symptoms until 5 or 6 months, with pain or swelling in the fingertips.

Along with pain, the risks for heart and kidney damage and stroke are increased.

She says that's why it's important to work with patients early on and regularly.
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