UCSB Science Line

It would be great if there were a simple cure for
hemophilia. As you know, people with hemophilia
have blood that does not clot well. They can
have severe bleeding, even from a small injury.
Some diseases can be cured by killing the bacteria
that causes them. Some diseases can be prevented
with vaccines against viruses. But Hemophilia
is not caused by a germ.
Hemophilia is caused by a small problem in the
DNA (mutation). There are many proteins
involved in making our blood clot. If the recipe
for even one of these proteins is changed,
clotting may be slow, or less effective, or not
happen at all.There are actually many types of
hemophilia depending on which protein recipe
(gene) has a mutation. Safely giving a person
a missing gene is not easy. The gene has to get to
the right kind of cells without damaging the cells
and causing other problems, like cancer. No one
has found a solution to that problem yet.
Right now, there are only treatments, not
cures. A person with hemophilia might need
platelets or proteins called clotting
factors. Platelets are pieces of special blood
cells. They help to plug up a wound. Clotting
factors work with the platelets to make a better
plug. One treatment takes clotting factors from
the plasma of healthy people and gives them to
people with hemophilia. Clotting factors can also
be produced by other animals and given to people.
Other times, people get platelets from donors.
I am a platelet donor. Once every few weeks, I go
to our local blood center and spend an hour or two
reading while a machine takes my blood, separates
out the platelets, and returns the rest of the
blood to my vein. Some of the people who get my
platelets have hemophilia. Some have cancer.
Some may have had an accident or surgery that used
up all of their own platelets. Others have other
blood diseases. Sometimes they take plasma too.
They can get clotting factors from my plasma.
Most of the genes that help with clotting are on
the X chromosome. This means that males are much
more likely than females to have hemophilia.Can
you figure out why?
You may want to study physiology or genetics to
learn more about hemophilia.
Thanks for asking,
Answer 2:
Hemophilia is a blood disease that is caused
by a mutation in DNA. DNA or
deoxyribonucleic acid is the genetic code
found in almost all the cells in your body and
provides the information needed for your body to
function. One of these functions is the ability to
clot blood when you get a cut. Mutations
are changes that happen in the DNA letters A, T,
G, and C that make up the DNA code.
For example, a mutation might result in an A
instead of a G. A lot of times this is not a
problem but sometimes it can cause lots of
problems and result in a disease like
hemophilia.
Since the cause of hemophilia is an actual
change in your DNA it is not an easy fix. We
can treat people with hemophilia by giving them
medicine that helps them clot blood but right
now there is no cure. We would have to change
the genetic sequence of the person to permanently
cure them. We would need to introduce a non-
mutated, "correct" form of the specific gene that
is causing the hemophilia in order to cure
someone. There are also different forms of
hemophilia that result from mutations in different
genes.
Fortunately scientists have been developing
different ways to be able to help people with
problems with their DNA. They will hopefully
be able to replace mutated genes with healthy ones
in the future.
Answer 3:
This is a really complicated question because the
biology of diseases is naturally complex. Human
bodies are never totally isolated and always have
multiple factors that determine how they react
when something goes wrong. Hemophilia cannot be
cured because it is a genetic disorder. This
means that the problem comes from an error in a
person’s DNA. Since every single cell in a
person’s body has their own copy of DNA, every
single cell has the same disorder. Repairing
broken or incorrect DNA is really hard for
scientists because DNA is protected and guarded in
the cell’s nucleus. Although scientists have a
few ways to alter or rewrite DNA, the technique
isn’t perfect enough to be used on humans yet.
Furthermore, it would be almost impossible to edit
every single cell in a person’s body to eliminate
the disorder entirely. This idea is really
complicated but considering ideas like these are
the first steps to becoming scientist! Thank
you for the question!
Answer 4:
It's genetic. People with hemophilia lack a
protein that causes blood to clot. Their
bodies cannot make this protein because the copy
of the gene that they carry and which is
responsible for this protein is defective. Because
it's not possible currently to alter a person's
DNA in a controlled fashion, hemophilia is
incurable.
In the future, it may be possible to insert a
working copy of the gene onto a virus and then use
the virus to implant the working gene into the
patient's genome. This would cure hemophilia and a
number of other bad genetic diseases.
Answer 5:
Hemophilia is a genetic disease that results
in uncontrolled bleeding after an injury.
Usually when someone gets a cut, the blood vessel
will get blocked which is called clotting.
This stops the blood from flowing out of the cut.
Blood clotting is a very complex process that
involves a lot of parts. In hemophilia, one of
those parts doesn’t work right so the entire
clotting process doesn’t work. To treat people,
you can give them the broken parts of the clotting
process, but they need to be given really
often. People are generally born with hemophilia
and get it from their parents. The best way to
prevent hemophilia is to fertilize the embryo
outside the body, check it for hemophilia, and
then implant it in a women’s uterus. This is not
generally how children are conceived so for the
most part the disease can’t be prevented.
The only way to cure the disease would be to
teach the body how to make the right parts of the
clotting process. This is called gene
therapy and involves genetically engineering a
person. Gene therapy is not generally used since
it’s still being developed and some people don’t
like the idea of changing a person’s genes.
Although, someday many genetic diseases may be
cured with gene therapy.