8 дек. 2023 г. · The US Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment ...

In conclusion, gene-corrected hematopoietic stem cell transplantation has promising outcomes for SCD, Fanconi anemia, and thalassemia, and it may overcome the ...

30 нояб. 2023 г. · The therapy, called Casgevy, has been approved for use to treat sickle cell anaemia and beta thalassemia, inherited, lifelong conditions that can be fatal.

Gene therapy by either gene insertion or editing is an exciting curative therapeutic option for monogenic hemoglobin disorders like sickle cell disease and ...

19 дек. 2023 г. · The goal of genetic therapies for SCD is to treat or cure the disease by changing DNA or adding new DNA. With Casgevy, the patient's blood stem ...

7 дек. 2023 г. · The Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel), ...

24 июн. 2022 г. · Gene therapy may treat SCD by editing DNA in hemoglobin genes to stop the disease. It can be done by either fixing the faulty hemoglobin gene or ...

13 дек. 2021 г. · An experimental gene therapy for sickle cell disease restored blood cells to their normal shape and eliminated severe pain crises for years ...

Gene therapy (GT) has been reported to improve bone marrow function in individuals with Fanconi anemia (FA); however, its clinical application is still in ...

26 сент. 2024 г. · Researchers in clinical trials are currently studying gene therapy as a potentially curative treatment for sickle cell disease.