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Gene Therapy

WHAT IS GENE THERAPY? HOW DOES IT WORK?

Gene therapy is the modification of the genome - the introduction, removal, or alteration of a gene to treat or prevent a condition or disease. This was the holy grail for medicine for decades and, with the development of safe and efficacious delivery systems, this is now a reality. A delivery system, also known as a vector, is a way to put the new material into the cell. 

Cats, dogs, horses, and human beings suffer from an increasing number of ailments as they get older. The development and progression of these diseases are driven by the aging process in people and animals. By addressing these directly through gene therapy, IHS Pets aims to rejuvenate your companions. 

Since the effects of gene therapy can be long lasting, treatments like Luxterna, an FDA approved gene therapy for inherited retinal diseases, only needs to be given once. 

Luxturna uses the same AAV vector as Integrated Health Systems. Delivering healthy copies of the RPE65 gene restores vision. Recognizing the vast number of applications, many researchers have begun to explore the technology’s potential. 

As of March 2019, 372 gene therapy clinical trials are underway. 


HOW GENE THERAPY WORKS

Sometimes the whole or part of a gene is defective or missing from birth, or a gene can change or mutate during adult life. Any of these variations can disrupt how proteins are made, which can contribute to health problems or diseases.

In gene therapy, scientists can do one of several things depending on the problem that is present:

  • They can replace a gene that causes a medical problem with one that doesn’t

  • Add genes to help the body to fight or treat disease

  • Or turn off genes that are causing problems

In order to insert new genes directly into cells, scientists use a vehicle called a “vector” which is genetically engineered to deliver the gene.

Viruses, for example, have a natural ability to deliver genetic material into cells, and therefore, can be used as vectors. Before, a virus can be used to carry therapeutic genes into human cells. However, it is modified to remove its ability to cause an infectious disease.

Gene therapy can be used to modify cells inside or outside the body. When it’s done inside the body, a doctor will inject the vector carrying the gene directly into the part of the body that has defective cells.

In gene therapy that is used to modify cells outside of the body, blood, bone marrow, or another tissue can be taken from a patient, and specific types of cells can be separated out in the lab. The vector containing the desired gene is introduced into these cells. The cells are left to multiply in the laboratory, and are then injected back into the patient, where they continue to multiply and eventually produce the desired effect.

WHAT ARE CELLS AND GENES? HOW DO THEY INTERACT?

Cells are the basic building blocks of all living things; the human body is composed of trillions of them. Within our cells there are thousands of genes that provide the information for the production of specific proteins and enzymes that make muscles, bones, and blood, which in turn support most of our body’s functions, such as digestion, making energy, and growing.

How our gene therapy works

Gene therapy offers the ability to permanently correct a disease at its most basic level, the genome. And could offer cures for many conditions that are considered incurable at this time. It is a flexible treatment option and gene therapy has the potential to treat a variety of illnesses by:

  • Replacing missing or defective genes which can cause inherited or acquired disorders.

  • Delivering genes that add needed proteins to the body.

  • Delivering genes that enhance the body to resist disease and perform at higher levels.

  • Introducing genes which stimulate cell growth and heal damaged tissue.

Gene therapy offers patient alternatives to regular daily maintenance. Patients suffering from life-threatening disease are often forced to endure swallowing pills or daily/weekly injections to treat and/or monitor their disease. Gene therapy could ease the burden of enduring the disease by offering the promise of 1-2 treatment injections in a patient’s lifetime with no need for long-term follow-up.

With the remarkable progress in genetics, we believe gene therapy will prove to play an increasingly prominent and transformative role in medicine. Gene therapy has the potential to treat and maybe cure monogenic diseases. Most monogenic diseases are ones for which a protein is defective or not being expressed. As such, gene therapy has the potential to address many of these congenital disorders. There is also a broad opportunity to apply gene therapy for acquired diseases as diverse as HIV, heart failure, Alzheimer’s and more.

 Past Success with Gene Therapy 

Gene therapy in animals has a robust record. A joint research project between Kazan University and the University of Nottingham found direct injections into affected limbs and ligaments can cure lameness in horses. 

Gene therapy has been used with success to treat hemophilia and muscular dystrophy in dogs.  

An article in Nature demonstrated that gene therapy for Sandhoff disease in cats resulted in “widespread correction of the central nervous system”

Although still under development, IL-12 gene therapy for targeting tumors has been called “safe and effective” in cats, dogs, and horses. More broadly speaking, gene therapies for treating and, one day perhaps preventing, cancer are not far away. 

Our therapies target a wide range of disorders by getting at the root cause.