Therapy FAQs

Many of the things about us are passed on from one generation to another through genes.

What are genes?

  • Many of the things about us are passed on from one generation to another through genes. Genes play an important role in how we look – curly or straight hair, short or tall, brown or blue eyes – and how our body works. They carry information that makes us who we are.
  • You have 2 copies of each gene, 1 inherited from each parent. Some genetic conditions happen when both parents pass on a dysfunctional copy of a specific gene to the child, and others happen when a child gets 1 non-functional copy. For hereditary angioedema (HAE) Types I and II, 1 non-functional copy of a gene from 1 parent is enough to cause the condition.

How do genes, DNA, and proteins fit together?

  • Genes are made up of DNA, which provide specific instructions (like a recipe) for making proteins in each of our cells. Proteins are the building blocks for everything in our body like bones and blood.
Illustration of the DNA, Protein and Parts of the Body

What does gene therapy mean?

  • Gene therapy is a potential therapy option that involves the transfer of a gene to replace a missing or dysfunctional gene.
  • Gene therapy is currently being tested for various diseases and conditions like hemophilia, phenylketonuria (PKU), and hereditary angioedema (HAE).

How is gene therapy designed to work?

  • There are different types of gene therapy being investigated like changing the DNA or introducing a new gene to the body.
  • In one type of gene therapy, a new functional gene is introduced to the body. The new functional gene is put into a vector, or viral shell, to deliver the gene to the body. A neutralized virus is often used as the vector, or vehicle. It is usually injected directly into a person’s vein as an intravenous (IV) infusion.
  • Once inside the cells, the functional gene is designed to produce proteins. The functional gene is not intended to be passed down to future generations.

What are the possible risks and benefits of gene therapies?

Possible risks:

  • You may have side effects or feel badly from the gene therapy. Side effects may range from mild to severe. The study doctor/staff may give you treatment to help your side effects improve or go away. In some cases, side effects can be serious, and some can last for a long time or never go away. There may be unknown side effects that could occur.
  • After receiving the gene therapy with the neutralized vector, your immune system might see the vector as a virus and remove it from your body. You may not be able to receive gene therapy again using the same vector or other types of vectors.
  • The vector may show up in your bodily fluids, like saliva, urine, stool (poop), and semen (sperm), and other people who you may have contact with. It is not fully understood how quickly the vector will disappear from your body fluids. For this reason, you should not donate blood, semen, eggs, tissues, or organs while participating in a clinical research study.
  • If you experience desired effects from the gene therapy, it is unknown how long the effects will last.

Possible benefits:

  • The intention of a gene therapy study is to introduce a functional gene to make the desired functional protein.
  • You may find that the gene therapy helps you feel better or lessens the symptoms of your condition or disease.
  • There is no guarantee that you will benefit from gene therapy. However, by participating in a gene therapy study, you may help researches learn more about a condition and how to treat it, which could also help others with that same condition in the future.