Strimvelis is a gene therapy consisting of a patient's own CD34 positive cells, transduced with a retroviral vector carrying the human ADA gene, and was the first ex vivo autologous gene therapy approved by the European Medicines Agency. It treats adenosine deaminase deficiency-severe combined immunodeficiency, known as ADA-SCID, a rare genetic disorder in which patients lack the enzyme adenosine deaminase needed for a healthy immune system and, without effective treatment, rarely survive more than two years. Strimvelis is used specifically for ADA-SCID patients who lack a matched stem cell donor, extracting, modifying and reinfusing a patient's own cells to restore immune function.
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Strimvelis
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Which patients is Strimvelis used for?
It is used for ADA-SCID patients who have no suitable matched related donor for a bone-marrow transplant.
Strimvelis is used in patients with ADA-SCID who cannot be treated by a bone-marrow transplant because they do not have a suitable, matched, related donor.
How is Strimvelis made from a patient's own cells?
It is made from CD34+ cells extracted from the patient's own bone marrow.
To make Strimvelis, a sample of the patient's bone marrow is collected. Then CD34+ cells, which are cells that can make lymphocytes, are extracted from the bone marrow cells.
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Source Strimvelis (Wikipedia)
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1. Wikipedia
Wikipedia contributors, Wikimedia Foundation 2. Strimvelis (Wikipedia)
- Treatment developed through a 2010 collaboration with Fondazione Telethon and Ospedale San Raffaele, History section
https://en.wikipedia.org/wiki/Strimvelis, lead section
Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence, sold under the brand name Strimvelis, is a medication used to treat severe combined immunodeficiency due to adenosine deaminase defici
Treats: Severe Combined Immunodeficiency, Lead: treats Severe Combined Immunodeficiency
Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence, sold under the brand name Strimvelis, is a medication used to treat severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID).
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