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DID YOU KNOW?

In the year 2017, there was an estimated 1,688,780 new cancer cases and along with that was a projected 600,920 that would lose their battle to the fatal illness in the U.S.

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The road to healing starts now
AND THAT HOPE IS GENE THERAPY

WHAT

What is gene therapy?

Gene therapy is an alternative to drugs or surgery. It is when genes are placed into the cells or tissue of the patient in order to treat an illness, but it can also be used to replace a damaged gene with a functional one. So as of now, it is a promising option to treat genetic disorders or fatal diseases.

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But there are two types of gene therapy:

  • Somatic Gene Therapy- This type of gene therapy is when the new genes getting inserted is inserted into cells that do not produce sperm or egg. This method will prevent the effects of the treatment from being passed down to the following generations.

  • Germline Gene Therapy- In contrast to somatic gene therapy, this method will have the new genes placed into cells that do produce sperm or egg. Therefore, the effects of the treatment will be passed on to the patient's children.

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HOW

How does gene therapy work?

Doctors will use a vector, which is a carrier molecule, to deliver the genes to the damaged cells.

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Gene therapy can work in multiple ways:

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       In the body therapy

  • Virus (Viral Vector) - For this method, doctors will use a virus to deliver the new genes to the targeted cells in the patient. How they do this is by first removing the genes in the virus that causes the illness. Afterwards, they will insert the new genes into the virus and inject them into the patient. Some viruses that doctors can use are retroviruses, adenoviruses, adeno-associated viruses, and herpes simplex viruses.​

  • Liposomes (Non Viral Vector) - Now this is a non-viral vector that is considered safe because there is no potential of it causing any disease. Again, they will place the new genetic material inside and inject it into the targeted cells in the patient.

      

       Out of the body therapy

  • Ex-Vivo Gene Therapy- For this method, doctors will either remove blood or bone marrow from the patient. Continuing on from there, they will separate out the immature cells. Once it has been separated, the new genes will be placed in them and inserted into the bloodstream. Then, the cells will proceed to the bone marrow where it will mature, multiply and replace the faulty cells. 

  

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WHEN

When did gene therapy begin?

The idea of gene therapy came to life around the 1960s and 1970s. It all began back in 1970 when Stanfield Roger advanced the concept of damaged DNA being replaced with effective DNA aka gene therapy. This concept was later published in an article titled "Gene therapy for human genetic disease?" written by Theodore Friedmann and Richard Roblin.

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It wasn't until September 14, 1990 at NIH Clinical Center that gene therapy was actually used in a patient. It was used in a 4 year old girl diagnosed with adenosine deaminase (ADA) deficiency, which is a genetic disorder that stopped her from being able to fight off infections. The doctors removed her white blood cells and implanted effective genes within them. Then that was injected back into her.

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WHY

Why is gene therapy used?

Gene therapy is used in the hopes that one day it will be able to prevent or cure disease without the use of drugs or surgery. Gene therapy may one day in the future eradicate and prevent hereditary illnesses as well as possibly be a cure for heart disease, AIDs, and cancer. But more than that, gene therapy could also be used to improve an immune system. There are some cases where the immune system fails to fight against diseased cells, so doctors can use gene therapy to train it to recognize threatening cells. 

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Gene therapy has multiple uses:

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  • replace damaged genes

  • transport genes into the body that

       helps to quicken destroy cancer cells

  • deliver genes that can make cancer

       become normal cells again

  • provide vaccination using bacterial

       or viral genes

  • supply genes that can help to grow

       new tissue

  • transfer genes that can assist in healing

       impaired tissue 

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But gene therapy can cure so much more than cancer

When thinking of gene therapy, most people tend to think of cancer. But it has the potential to treat cystic fibrosis, hemophilia, heart disease, Parkinson's, and many more.

Click below to discover more

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