WHAT IS GENE THERAPY?
Imagine that you accidentally broke one of your neighbor's windows. What would you do? You could:
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?
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.
Genes, proteins and genetic disorders
Want to review more background on genetic disorders? Visit the Genetic Disorder Corner.
Need basic information about genes and proteins? Check out The Basics and Beyond.