What is Gene Therapy For Hemophilia?

By on July 9, 2013

About Hemophilia:

Gene therapy for hemophilia! Before knowing about gene therapy, you should know about hemophilia.

Hemophilia is a bleeding disorder caused by a genetic defect in a pair of chromosomes. The bleeding disorder occurs because of certain blood clotting factors such as VII and IX are missing and not working properly.

Most of these blood clotting proteins are produced in the liver. There are two forms of hemophilia such as A and B. The two major forms of hemophilia occur more commonly in men than in women.

Gene Therapy For HemophiliaIf you have hemophilia, you may bleed more easily and severely after an injury or cut compared to normal people. Sometimes, you can have internal bleeding in joints and muscles such as elbow, knee, ankle and hip.

Continued bleeding into the muscles and joints will cause permanent damage like joint problems and reduced mobility. But rarely this disease leads to death because of internal bleeding into the brain and other important organs.

Symptoms of hemophilia:

The symptoms of hemophilia may vary from person to person, based on the clotting factor in your blood.

The signs of hemophilia may include bleeding in urine and stool, changes in behavior, continuous bleeding after the umbilical card is cut at birth, neck pain, vomiting, excessive sleepiness, long-term headaches, pain in the joints and double vision.

If you want to treat these symptoms, there are many treatments available, but a gene therapy for hemophilia is one of the best and successful treatments.

When to consult a doctor?

If you have symptoms of hemophilia, your doctor will do a physical examination to find out the type of hemophilia and he or she will ask about your family history as this disorder tends to run in the families.

Your doctor will suggest some blood tests to determine how much clotting factors present in your blood.

Is gene therapy for hemophilia work best?

Nowadays, many doctors are suggesting gene therapy for hemophilia as it is a more successful and powerful method. Gene therapy is a technique for correcting the defective genes which are responsible for disease development.

Gene therapy is mainly designed to introduce new genetic material into cells to compensate the normal copy of the gene to restore the function of the protein. A new gene is injected into an adenovirus vector to change the DNA of the virus.

This virus is used to introduce the changed DNA into human cell, which helps to boost the production of the blood clotting factors.

A single dose of gene therapy boosts the production of missing blood-clotting factors in people with hemophilia. This therapy may give a permanent solution for preventing serious bleeding episodes.

These new technologies will be used to destroy the viruses in the blood and also successful in eliminating the risk of contracting HIV or hepatitis C from clotting factors.

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