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Gene therapy. Diseases such as sickle cell disease for which a person's normal phenotype or cell function may be restored in cells that have the disease by a normal copy of the gene that is mutated, may be a good candidate for gene therapy treatment. The risks and benefits related to gene therapy for sickle cell disease are not known.
Sickle cell is the most common disease to receive approval for gene therapy treatment, following decades of development and years of approvals for therapy for people with rarer conditions.
DB15572. UNII. S53L777GM8. KEGG. D12749. Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease [1] [3] and transfusion-dependent beta thalassemia. [1] It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease.. The most common side effects include stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with chemotherapy and ...
Conversations around sickle cell gene therapies should lead with the notion of how effective the therapies will be for patients rather than the cost, because it changes the “focus and the ...
Experts suggest Britain’s approval is a sign a U.S. authorization was likely “imminent” for sickle cell therapy LONDON (AP) — […] The post The world’s first gene therapy for sickle ...
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