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.
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
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
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: www.hemophilia.org and a World
Federation of Hemophilia: www.wfh.org
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