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On this page:
Definition of Gene Therapy:

Gene therapy is an intervention in which the gene that is mutated in affected individuals is augmented by the introduction of a functional version of the gene. The gene can be introduced as free DNA, in a lipid coat (liposome) or as part of a viral vector. The latter is the most common way of introducing genes and it involves modification of a specific virus so that it cannot cause disease and then having it carry the gene for the missing enzyme to the brain or any other organ of interest.

Taken from the Therapeutic Approaches page on the NTSAD website.


This is a link to KIDSHEALTH.ORG: The Basics of Genes and Genetic Disorders.  It's made for kids and it has audio which makes it easier to get through (but it's still kind of boring).

This is a link to HUMAN GENOME PROJECT INFORMATION.  This page answers the following questions.
Back to top

The following video shows the results of
gene therapy in mice:

 Tay-Sachs Gene Therapy Consortium

stompsf | October 17, 2010

Gene therapy studies of direct injection of AAV vectors into the brain of GM2 ganglia cytosis mice, which mimic Tay-Sachs disease in humans.

Category: Science & Technology
Tags: Tay-Sachs

 What is Gene Therapy

This following is a copy of one page of the Learn.Genetics website from the University of Utah. It is an interactive website that is comprehensive and easy to follow.

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.

This page was last updated on 05/04/2010    15 North 2030 East, Salt Lake City, Utah 84112-5330, (801) 585-3470   
Cite this page

Genetic Science Learning Center (2010, May 4) What is Gene Therapy?. Learn.Genetics. Retrieved May 4, 2010, from


 Biotechnology & Gene Therapy

This is a YouTube Video on Biotechnology and Gene Therapy.  It's long, but if you pay attention, it's much easier to learn about gene therapy this way than by reading long, boring articles.

dremchyld December 16, 2007Project for Biology 111. 3dB. Wed Pm. Biotechnology Project on Gene Therapy.
Rebecca Proctor, Chiquetta Silver, and Shamonia Wright.

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