Gene therapy (transfer or addition) Gene therapy (transfer or addition) The transfer or addition of genetic material into a cell. Once in the cell, a working copy of a gene can help make proteins despite the presence of a faulty or mutated gene. doesn’t just aim to treat symptoms of a disease or disorder. Instead, gene therapy can potentially help treat certain diseases by introducing a healthy (working or functioning) gene that may improve the burden of hemophilia treatment.
In the case of hemophilia A or B, the introduced gene is intended to inform the body to produce the factor VIII or factor IX protein involved in blood clotting.
Gene therapy
research in
hemophilia
For more information on who has been included in gene therapy research so far, click here.