UCSB Science Line
Sponge Spicules Nerve Cells Galaxy Abalone Shell Nickel Succinate X-ray Lens Lupine
UCSB Science Line
Home
How it Works
Ask a Question
Search Topics
Webcasts
Our Scientists
Science Links
Contact Information
Why cant their be a cure for hemophilia?
Answer 1:

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.



Click Here to return to the search form.

University of California, Santa Barbara Materials Research Laboratory National Science Foundation
This program is co-sponsored by the National Science Foundation and UCSB School-University Partnerships
Copyright © 2015 The Regents of the University of California,
All Rights Reserved.
UCSB Terms of Use