I am not a hemophilia researcher but I do work
with blood- from fish! I do know that one
important area of research on hemophilia is gene therapy. Gene therapy is a type of treatment where researchers and doctors can actually repair genes that have been damaged.
Since hemophilia is caused by damage or "mutations" on one single gene, it could be cured if researchers can find a way to
replace the defective gene with a normal gene. These normal genes will be carried into the body using a vector which is like a vehicle for transporting the gene into the cell.
Most vectors are harmless viruses that are able to insert the new DNA (or normal gene) into the cells. The virus "infects" cells with the healthy gene, overriding the hemophilia gene which causes the illness. In clinical trials on lab animals, gene vectors have been used to cure hemophilia A and B. The body's immune system does not appear to release antibody inhibitors when gene therapy is used.
Future gene therapy clinical trials will involve human subjects. Hemophilia researchers need to determine if the new gene can be passed from parents to their children or if they would have to insert the good DNA into the
cells of each generation. One potential problem is
that the new healthy gene contains extra
information. This extra information is required to make the healthy gene work after it "infects" cells. But it is not known how this extra genetic material will function in future generations.
Hemophilia clinical trials are exploring the
possibility that a patient's cells can be infected in the laboratory and then reintroduced to the body, thereby removing the chance of extra, unwanted, genetic material being passed on to future generations.
Gene therapy research may produce a cure for hemophilia but there is a lot more research that needs to be done. This type of research would be funded by a number of different government and private organizations like the National Institutes of Health (NIH), Centers for Disease Control and Prevention (CDC), The Coalition for Hemophilia B, the National Organization for Rare Disorders (NORD) and the Agency for Healthcare Research and Quality (AHRQ) to name a few.
There is a National Hemophilia Foundation (NHF) where you can get lots of information and a World Federation of Hemophilia: WFH.
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