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gene therapy

What Is Gene Therapy?
Image: iStockphoto.com/dra_schwartz

What Is Gene Therapy?

For people with serious genetic illnesses, gene therapy offers hope. The promise of gene therapy is to insert properly functioning genes into the body to cure or treat the problem.

Learn more about how gene therapy works, who could benefit, and the ethical issues it raises. Follow the links for games, activities, and more.

University of Utah
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Haemophilia Gene Therapy Breakthrough

Haemophilia Gene Therapy Breakthrough

Working with colleagues at St. Jude's Children's Research hospital, Dr. Amit Nathwani's promising research may one day help treat hemophilia. More...

Profile
UCL Cancer Institute
‘Bubble Kid’ Success Puts Gene Therapy Back on Track

‘Bubble Kid’ Success Puts Gene Therapy Back on Track

Five babies with a rare condition have healthy immune systems thanks to a recent gene therapy treatment. Read more about it, and follow the timeline of gene therapy success and failure. More...

Article
New Scientist
A New Gene Therapy, an Old Hard Truth

A New Gene Therapy, an Old Hard Truth

Gene therapy offers great hope, but medical research is full of unknowns. Read the story of this family's long struggle to treat a rare disease with gene therapy. More...

Article
National Geographic
Fact Sheet: Gene Therapy

Fact Sheet: Gene Therapy

What is gene therapy and how can it be used? Click the link on this page to get the answers. More...

Article
NSW Government
Gene Therapy Emerges from Disgrace to Be the Next Big Thing, Again

Gene Therapy Emerges from Disgrace to Be the Next Big Thing, Again

The tragic death of an 18-year-old in a 1999 gene therapy trial all but halted gene therapy research. Now with researchers' new understanding, gene therapy may again be on the rise. More...

Profile
WIRED
Mouse Race: Discovering Exercise in a Pill

Mouse Race: Discovering Exercise in a Pill

Race these electronic mice in a virtual experiment to learn how drugs that mimic exercise were found and how they could work. More...

Interactive
NOVA/PBS
New Genetic Therapy Could Erase Down Syndrome

New Genetic Therapy Could Erase Down Syndrome

Chromosome therapy, essentially gene therapy on steroids, may “cure” Down syndrome. There may be a way to turn off, or silence, the entire extra chromosome. More...

Article
WBUR
Talking Glossary of Genetic Terms

Talking Glossary of Genetic Terms

Dr. Fabio Candotti defines gene therapy and explains how it can be used. Don't miss the figure that helps explain the concept. More...

Interactive
Genome.gov
Understanding Gene Therapy

Understanding Gene Therapy

Sometimes gene therapy doesn't need to be permanent. This gene therapy treatment could trigger growth of new, clear arteries in heart patients…by genes working in the body for just a few weeks. More...

Video
HowStuffWorks
What Is a Chromosome?

What Is a Chromosome?

Many of the things that make you you are the result of what's in your genes. To understand more about how heredity works, take this tour of the basics. More...

Interactive
University of Utah

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