ZYNTEGLO™ is a one-time gene therapy that addresses beta-thalassemia at the genetic level
ZYNTEGLO uses your own blood stem cells to treat beta-thalassemia
Blood stem cells are collected from your body
- ZYNTEGLO is made specifically for you using your own cells
- Once collected, your blood stem cells are sent to a lab to begin manufacturing
ZYNTEGLO is manufactured by adding working copies of the beta-globin gene to your collected cells
- The working gene is added to your collected blood stem cells with help from a vector outside of your body
- Learn more about vectors below
Your modified stem cells (ZYNTEGLO) are returned to your body through an intravenous (IV) infusion
- Blood stem cells move through your bloodstream to your bone marrow
- These blood stem cells then produce new blood stem cells in a process called engraftment
New red blood cells are produced containing functional adult hemoglobin
- New cells contain working copies of the beta-globin gene leading to the production of functional adult hemoglobin
What is a vector?
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A vector is like an envelope that delivers the working copies of the gene directly to the “address” of your blood stem cells -
ZYNTEGLO uses a type of vector called a lentiviral vector (LVV). The LVV uses HIV’s natural ability to deliver genes into a cell but does not include the genes that cause HIV infection -
The LVV is built using only the parts of HIV that are good at delivering the working copies of the beta-globin gene to your stem cells -
The working copies of the modified beta-globin gene are delivered by the vector directly into your blood stem cells
Important Considerations
Although ZYNTEGLO will not give you HIV infection, treatment with ZYNTEGLO may cause a false-positive HIV test result by some commercial tests. If you need to have an HIV test, talk with your doctor about the appropriate test to use.
HIV = human immunodeficiency virus.
Talk with your doctor about ZYNTEGLO
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