Gene Therapy

1 What is a Gene Therapy?

A treatment which involves altering the genes inside your body’s cell to stop the disease is called gene therapy.

Our genes contain our DNA which is the one that controls your body’s function and forms from regulating your body systems to making you grow taller.

Diseases can re a result of genes that does not work properly. To improve your body’s ability to fight disease or in an attempt to cure disease, gene therapy will replace a faulty gene or will add a new gene.

There is a wide range of diseases such as cancer, cystic fibrosis, diabetes, heart disease, AIDS and hemophilia that gene therapy can treat.

This therapy is available only as part of a clinical trial in the United States because researchers are still studying how and when to use gene therapy.

2 Reasons for Procedure

Here are the most common reasons to undergo a gene therapy.

In order to help your body better fight disease or to cure a disease, gene therapy will be used to correct defective genes.

Researchers are investigating several ways to do this such as:

Fixing mutated genes – healthy genes that prevent disease can be turned on to inhibit the disease or mutated genes that cause disease will be turned off.

Replacing mutated genes – this may help treat certain disease because certain genes no longer work at all, for example, a gene called p5 normally prevents tumor growth in your body but if doctors can replace this defective gene it may trigger the cancer cells to die

.Making diseased cells more evident to the immune system – some of the diseased cells will not be attacked by your immune system because it does not see them as intruders, your doctors will use this therapy to train your immune system to recognize the cells that are a threat.

3 Potential Risks

Gene therapy has some possible risks. A carrier called the vector has to deliver the gene because genes cannot be directly inserted into your cells.

The most common vectors are viruses because they can recognize certain cells and carry genetic material into the gene cells.

Some of the possible risks include:

  • unwanted immune system reaction – this may cause inflammation or organ failure because the newly introduced viruses will see as intruders by your immune system
  • targeting the wrong cells – this can cause illness or disease even if cancer because viruses can affect more than one type of cells which can lead to the altered viruses infecting additional cells
  • infection caused by the virus – the viruses may recover their original ability to cause disease
  • the possibility of causing a tumor – is the new genes are inserted in the wrong spot in your DNA

To ensure the safety of those who are participating in the studies, the Food and Drug Administration and the National Institutes of Health are closely monitoring the clinical trials under way in the U.S.

4 Preparing for your Procedure

Required preparation for your gene therapy includes:

  • your doctor must find the faulty gene that causes a specific condition to identify it
  • he will locate the affected cells in the body’s organs or tissues
  • a working version of the gene must be available
  • then he will deliver this gene to the cell

5 What to Expect

Here you can find out what to expect from your gene therapy. To participate in a clinical trial is the only way to receive a gene therapy.

Clinical trials are research studies that help doctors determine if a gene therapy is safe for people and at the same time this can help doctors understand the effects of gene therapy.

The procedure will depend on the disease that you have and the type of gene therapy that being used. Your doctor may remove a bone marrow from your hipbone or he may draw blood from you.

These bone marrow and blood will be exposed to the virus or another type of vector that contains the desired genetic material.

After the vector has entered the cells, these cells will be injected back into your body into a tissue or vein along with the altered genes.

Other vectors that are being studied include:

  • stem cells – this can be trained to become cells that can help fight disease
  • liposomes – these are fatty particles that can carry new genes to pass the genes into your DNA cells

6 Procedure Results

If you do not understand your gene therapy results, consult with your doctor. There is still little evidence of the possibilities of gene therapy.

Clinical trials of this therapy in people have shown some success in treating:

  • leukemia
  • severe combined immune deficiency
  • blindness caused by retinitis pigmentosa
  • hemophilia

But there are barriers that stand in the way of gene therapy becoming a reliable form of treatment such as:

  • finding a reliable way to get genetic material into cells
  • targeting the correct cells
  • reducing the risk of side effects

This is still an active research nowadays.

7 Related Clinical Trials