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Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease. [1] [3] [4] [5]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 ...
The two gene therapies are the first approved in the U.S. for sickle cell disease. The FDA has previously OK’d 15 gene therapies for other conditions. In the U.S., an estimated 100,000 people ...
Sickle cell patient Dominique Goodson, 38 and pregnant with her first child, said she would like to get a gene therapy a year or so after she gives birth in December, but she needs to make sure ...
The US Food and Drug Administration on Friday approved two gene-based treatments for sickle cell disease, including the first therapy that uses the gene-editing technique CRISPR, opening a new era ...
In people with sickle cell trait, only one gene is abnormal; erythropoiesis generates a mixture of normal HbA and sickle HbS. The person has very few if any symptoms of sickle cell disease but carries the gene and can pass it on to their children. [56] Autosomal recessive inheritance means acquiring two changed genes from each parent. If both ...
Sickle cell disease (SCD) is a painful, inherited blood disorder in which the body makes flawed, sickle-shaped hemoglobin, impairing the ability of red blood cells to properly carry oxygen to the ...
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