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Gene Therapy

Written By Monica Savaglia

Posted July 28, 2016

Cancer is the leading cause of death worldwide… for about 8 million people. 

It’s likely that you’ve known someone with cancer, or maybe you’ve even been diagnosed with some form of cancer.

The National Cancer Institute reported that this year, an estimated 1,685,210 new cases of cancer will be diagnosed in the U.S., and 595,690 people will die from the disease.

Fighting cancer isn’t easy for anyone involved. It’s emotionally and physically grueling, and at a certain point during all of the treatments and surgeries, the idea of being cancer-free seems hopeless.

But what if there was a treatment that could cure a life-changing disease like cancer?

Well, there is.

What is Gene Therapy?

Currently, clinical trials are being conducted on a treatment that fights against cancer and other genetic diseases, and it’s called gene therapy.

Gene therapy is the transplantation of normal genes into cells in place of missing or defective genes in order to correct genetic disorders.

The most common way for a normal gene to travel into the body is by a carrier, also known as a vector.

The vector is usually a virus because viruses can recognize certain cells and carry genetic material into the cells’ genes.

Essentially the new gene is knocking out the mutated gene that isn’t functioning properly, so the new gene can replace it and start fighting the disease.

The Advantages

A huge benefit of this treatment is its less invasive approach. Patients won’t have to undergo surgery or use drugs that could have a serious impact on their well-being.

It’ll have the ability to offer patients a long-term outcome, with a strong probability that the disease will be cured with a single treatment.

The treatment would be ideal for inherited diseases, some types of cancer, and certain viral infections. Other diseases that would be candidates are: AIDS, cystic fibrosis, Parkinson’s, Alzheimer’s, Lou Gehrig’s disease, cardiovascular disease, and arthritis.

These diseases affect a huge portion of the world’s population. For example, 44 million people worldwide live with Alzheimer’s. reports that by 2050, more than 16 million Americans will have the disease unless a cure is found.

Genetic therapy could free these people and their families from the hardships of living with a life-altering genetic disease.

Companies That Are Leading the Way

Biotech companies uniQure N.V. (NASDAQ: QURE) and Celsion Corp. (NASDAQ: CLSN) are working hard at researching and conducting clinical programs so gene therapy can safely begin curing the many people who suffer from genetic diseases.

uniQure is a leader in gene therapy right now because of its ambitious and dedicated work to ensure treatment is safe and effective for patients.

Specifically, it works on gene therapy treatments for diseases in the liver/metabolism, the central nervous system, and the cardiovascular system.

Here is what uniQure has in the pipeline for its research and clinical programs:

uniQure pipeline

Celsion is a fully incorporated oncology company whose focus is on developing its innovative cancer treatments. Among those treatments are its gene therapy platforms called TheraPlas and TheraSilence.

TheraSilence is currently in its research phase before it goes into “pre-clinical.” This platform focuses on delivering synthetically generated RNA-based non-viral carriers.

If this platform works out, Celsion will have a great advantage because the common procedure for gene therapy is to have the disease-free gene carried in a virus, which poses some risks to the patient.

The Disadvantages

Of course, with anything new and different, there will be people who don’t agree despite how great the outcome could be.

People who oppose gene therapy have the concern that if we start altering genes in any way, then it’ll get to the point where we’ll be choosing specific genes to create an “elite” group of humans.

If people have the ability to pick and choose specific genes, then they’ll become “creators” of humans rather than leaving it up to nature. Modifying humans to have elite genes would forever change the way we see human lives and the uniqueness of their creation.

Another concern is that gene therapy will be very expensive and it’ll only be available for the rich, while the poor won’t be able to afford it. This would be unfair, because who’s to judge if one person’s life is valued higher than someone else’s because of the amount of money they have?

The risks factors involved with gene therapy are:

  • The possibility of the body’s immune system reacting to the virus since it’s an intruder to the body.
  • The potential for the virus to recover its original ability to cause disease.
  • The risk of the new gene getting inserted in the wrong spot in DNA, which could result in the formation of tumor.

While there will always be risks involved when it comes to treating a disease, if you think about the outcome of gene therapy… it’s worth it. Especially if you’ve been living with a disease for most of your life… you’ll have hope again.

These risks will significantly reduce as gene therapy continues to be studied and researched closely to make sure its benefits outweigh the risk of more harm and illnesses.

Gene therapy is beginning to alter medicine and the way diseases are treated… and cured.