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Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease [3] [5] and transfusion-dependent beta thalassemia. [3] 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. [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 ...
In 2023, both exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia) were approved for the treatment of sickle cell disease. [ 12 ] [ 130 ] [ 14 ] Kendric Cromer in October 2024 became the first commercial case in the USA to receive gene therapy and was discharged from Children's National Hospital . [ 131 ]
Casgevy works by editing the DNA in a patient’s stem cells — which are responsible for making the body’s blood cells — so that they no longer produce sickle-shaped cells.
Other books on similar topics include A Treatise on the Calculus of Finite Differences by George Boole, Introduction to Difference Equations by S. Goldberg, [5] Difference Equations: An Introduction with Applications by W. G. Kelley and A. C. Peterson, An Introduction to Difference Equations by S. Elaydi, Theory of Difference Equations: An Introduction by V. Lakshmikantham and D. Trigiante ...
Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells.
Advances in Difference Equations is a peer-reviewed mathematics journal covering research on difference equations, published by Springer Open.. The journal was established in 2004 and publishes articles on theory, methodology, and application of difference and differential equations.
Casgevy works by editing the BCL11A gene, which normally inhibits the production of HbF in adults. The edit has the effect of increasing production of gamma globin, a component of fetal hemoglobin HbF, and thereby resolving the anemia.