What is Gene Therapy?

Imagine that you accidentally broke one of your neighbor's windows. What would you do? You could:

1. Stay silent: no one will ever find out that you are guilty, but the window doesn't get fixed.
2. Try to repair the cracked window with some tape: not the best long-term solution.
3. Put in a new window: not only do you solve the problem, but also you do the honorable thing.

What does this have to do with gene therapy?

You can think of a medical condition or illness as a "broken window." Many medical conditions result from flaws, or mutations, in one or more of a person's genes. Mutations cause the protein encoded by that gene to malfunction. When a protein malfunctions, cells that rely on that protein's function can't behave normally, causing problems for whole tissues or organs. Medical conditions related to gene mutations are called genetic disorders.

So, if a flawed gene caused our "broken window," can you "fix" it? What are your options?

1. Stay silent: ignore the genetic disorder and nothing gets fixed.
2. Try to treat the disorder with drugs or other approaches: depending on the disorder, treatment may or may not be a good long-term solution.
3. Put in a normal, functioning copy of the gene: if you can do this, it may solve the problem!

If it is successful, gene therapy provides a way to fix a problem at its source. Adding a corrected copy of the gene may help the affected cells, tissues and organs work properly. Gene therapy differs from traditional drug-based approaches, which may treat the problem, but which do not repair the underlying genetic flaw.

But gene therapy is not a simple solution - it's not a molecular bandage that will automatically fix a disorder. Although scientists and physicians have made progress in gene therapy research, they have much more work to do before they can realize its full potential. In this module, you'll explore several approaches to gene therapy, try them out yourself, and figure out why creating successful gene-based therapies is so challenging.

Supported by a Science Education Partnership Award (SEPA) [No. 1 R25 RR16291-01] from the National Center for Research Resources, a component of the National Institutes of Health, Department of Health and Human Services. The contents provided here are solely the responsibility of the authors and do not necessarily represent the official views of NCRR or NIH.