What is sickle cell anemia

A stem cell or bone marrow transplant is the only cure for sickle cell anemia. But they’re risky procedures, so they’re reserved for people with severe disease. Newer treatments like gene therapy may become an option in the future. Sickle cell anemia (SCA) is an inherited disorder that affects your red blood cells. It’s most common in people who live in or have ancestors from sub-Saharan Africa, India, Central or South America, or parts of the Mediterranean and Middle East. Also known as subtype HbSS, SCA is the most common type of When you have SCA, sickle cells can die prematurely, leaving your body with a red blood cell deficit. Due to their shape, they may also get stuck in your blood vessels. Combined, these two issues can lead to various health problems, some of which may be life threatening. The only cure for SCA is a stem cell or bone marrow transplant. But there are limitations to these procedures, which makes their use in SCA relatively uncommon. As scientists discover more about possible treatments for SCA, newer options may emerge. A doctor may consider a Your bone marrow is where red blood cells form. With Your bone marrow is also the site of stem cell production. These are early cells that later become red and white blood cells and platelets. As of January 2023, stem cell or bone marrow transplants are the only Food and Drug Administration (FDA) approved cures for SCA. In theory, a bone marrow or stem cell transplant could provide your body a fresh start with...

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Sickle cell anemia, a type of sickle cell disease (SCD), is a genetic condition that affects your red blood cells (RBCs). It affects around Healthy RBCs are donut-shaped. They’re flexible and can easily move through the tiniest of your blood vessels. However, if you have Sickle-shaped RBCs are prone to getting stuck in small vessels, making it hard for blood to reach many parts of your body. This can cause pain, infections, and tissue damage. Until recently, Because of these factors, current treatment options are often not possible or recommended for people with SCD. However, a new cure for SCD has recently appeared on the horizon: gene therapy. What is it, and when can you receive this treatment? Read on to find out. Each of your cells contains These instructions can sometimes have typos, or mutations. Most of the time, mutations aren’t of major consequence, but sometimes they can hit critical parts of your genes. This can harm your cells’ ability to properly perform their assigned task. This is what happens in SCD. Gene therapy uses specialized molecular tools called There are a couple of ways Gene editing In this approach, scientists use CRISPR-Cas9 to cut your DNA at the sites of the mutations and replace them with the “correct” code. This is called gene editing based on its similarity to the work of a book editor. When used in SCD, CRISPR-Cas9 edits mutations in your hemoglobin genes. This restores hemoglobin’s ability to capture oxygen and gives RBCs back their healt...