Can hemophilia be cured? It depends on the definitiondoi:10.1016/j.rpth.2024.102559ethicsgenetic therapyhemophilia Ahemophilia BOver the years, the palette of treatment options for hemophilia has grown extensiv
Providing a long-term cure of these diseases requires not only that the hepatocytes be efficiently transduced, but also that the vectors remain transcriptionally active and give rise to therapeutic levels of functional protein. We have recently demonstrated that hemophilia A could be cured in newborn...
The trial, sponsored by Akouos, Inc, a wholly owned subsidiary of Eli Lilly and Company, is one of several underway or about to start in the United States, Europe and China, where a handful of other children have already been reportedly cured. "As more patients at different ages are treat...
as a treatment for these two disorders. Other major genetic disorders like hemophilia and congenital blindness are curable through gene therapy. Acquired diseases are treated with different gene therapies. Such therapeutic strategies include gene vaccines and oncolytic virotherapy. However, for neurodegenera...