Sickle Cell Disease What Is Sickle Cell Disease?

Sickle cell disease — also called sickle cell anemia — is a group of

inherited

inherited

(in-HAYR-ih-ted):

The passing of genetic information from a parent to a child through genes.

disorders that affect

hemoglobin

hemoglobin

(HEE-moh-GLOH-bin):

A protein in red blood cells that carries oxygen throughout the body.

, the major protein that carries oxygen in red blood cells. Normally, red blood cells are disc-shaped and flexible so they can move easily through the blood vessels. In sickle cell disease, red blood cells are misshaped, typically crescent- or “sickle”-shaped due to a gene

mutation

mutation

(myoo-TAY-shun):

Any change in a nucleic acid sequence, such as DNA or RNA.

that affects the hemoglobin molecule. When red blood cells sickle, they do not bend or move easily and can block blood flow to the rest of the body.

Serious problems may come from having sickle cell disease, including sudden onset of strong episodes of pain, called pain crises, that occur because of blocked blood flow and often require medical attention Other terms used to describe these pain episodes include “sickle cell crisis” and “vaso-occlusive crisis.” People who have sickle cell disease may also experience other serious health complications, such as chronic pain, stroke, lung problems, eye problems, infections, and kidney disease.

Sickle cell disease is a lifelong illness that may be managed through preventive screening and treatment strategies. Treatment reduces or helps manage symptoms and increase longevity. With the right care, many people who have sickle cell disease live fulfilling lives and safely participate in most activities.

The NHLBI Drives Progress Toward New Therapies

In December 2023, the U.S. Food and Drug Administration approved two new gene therapies that are transformative therapies for sickle cell disease:

• One treatment adds a modified gene into the body.
• One treatment makes a change to a gene that is already in the body.

Sickle cell disease affects more than 100,000 people in the United States and 8 million people worldwide. In the United States, more than 90% of people who have sickle cell disease are non-Hispanic Black or African American, and an estimated 3%-9% are Hispanic or Latino.

• About 1 in 13 Black babies are born with sickle cell trait, meaning that they inherited a sickle cell gene from one parent.
• About 1 in every 365 Black babies are born with sickle cell disease, meaning they inherited a sickle cell gene from each parent.

The NHLBI leads and supports research and clinical trials to find more transformative therapies for sickle cell disease.

BOOKLET

Sickle Cell Disease: Milestones in Research and Clinical Progress Booklet

Learn about the history of sickle cell disease in the United States, from its discovery in 1910 to the NHLBI legacy of research that has advanced the understanding of sickle cell disease, improved clinical progress, and paved the way for widely available therapies.

Download the booklet

Book traversal links for Sickle Cell Disease

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Causes and Risk Factors

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