Targeted Gene Therapy

Also known as: targeted therapy

What is targeted gene therapy?

Targeted gene therapy is a form of cancer treatment that can actively treat and even destroy cancerous cells without impacting the body’s normal cells. Cancerous cells have genetic differences that distinguish them from normal cells in the body. These targeted therapies are designed to recognize these cancerous cells through their unique formulation of proteins or enzymes. Then they can act to halt their growth or even to destroy them.

What happens during the treatment?

Much like antioangiogenesis therapy, targeted gene therapy will be prescribed by a health care provider. Depending on which medication is indicated, you will take the medication by mouth as a pill or capsule or it will be administered direction into the vein as an intravenous solution. The specific dosage and timing of each dosage will vary based on the medications used.

Is any special preparation needed?

No special preparation is required before starting targeted gene therapy. The drugs do have some side effects, so you’ll want to talk to your doctor about the risks and benefits before starting the medication.

What are the risk factors?

The side effects of targeted gene therapy can include diarrhea, liver problems, fatigue, mouth sores, skin problems, high blood pressure, problems with blood clotting or wound healing and more. Occasionally more serious side effects can occur.

Reviewed by: Kristen Stabingas, MD

This page was last updated on: 12/30/2025 3:56:57 PM