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[8] [9] It was developed as a recombinant protein (rhIL-11) as the drug substance oprelvekin. The human IL-11 gene, consisting of 5 exons and 4 introns, is located on chromosome 19, [6] and encodes a 23 kDa protein. IL-11 is a member of the IL-6-type cytokine family, distinguished based on their use of the common co-receptor gp130.
When administered systemically, or in high doses, viral vectors may induce an inflammatory response, which can cause minor side effects such as edema or serious ones like multisystem organ failure. [30] It may also be difficult to administer gene therapy repeatedly due to the immune system's enhanced response to viruses.
Oprelvekin is recombinant interleukin eleven (IL-11), [1] a thrombopoietic growth factor that directly stimulates the proliferation of hematopoietic stem cells and megakaryocyte progenitor cells and induces megakaryocyte maturation resulting in increased platelet production.
This is one of the first studies of a CRISPR-based in vivo human gene editing therapy, where the editing takes place inside the human body. [266] The first injection of the CRISPR-Cas System was confirmed in March 2020. [267] Exagamglogene autotemcel, a CRISPR-based human gene editing therapy, was used for sickle cell and thalassemia in ...
Interleukin 11 receptor, alpha subunit is a subunit of the interleukin 11 receptor. IL11RA is its human gene. [5] Interleukin 11 is a stromal cell-derived cytokine that belongs to a family of pleiotropic and redundant cytokines that use the gp130 transducing subunit in their high affinity receptors. This gene encodes the IL-11 receptor, which ...
Its physicians also plan before the end of the year to use a newly approved gene-editing therapy to, for the first time, treat a Michigan person with sickle cell disease using another Lyfgenia or ...
Exagamglogene autotemcel is the first cell-based gene therapy treatment utilizing CRISPR/Cas9 gene editing technology to be approved by the US Food and Drug Administration (FDA). [13] The most common side effects include low levels of platelets and white blood cells, mouth sores, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile ...
See the list of FDA-approved therapeutic monoclonal antibodies in the monoclonal antibody therapy page. This is a dynamic list and may never be able to satisfy particular standards for completeness. You can help by adding missing items with reliable sources .