Gene Therapy

Explore the what's and why's of gene therapy research, includingan in-depth look at the genetic disorder cystic fibrosis and how gene therapy could potentially be used to treat it.

Explore the methods for delivering genes into cells.

You are the doctor! Design and test gene therapy treatments with ailing aliens.

Researchers hoping to bring gene therapy to the clinic face unique challenges.

Beyond adding a working copy of a broken gene, gene therapy can also repair or eliminate broken genes.

The future of gene therapy is bright. Learn about some of its most encouraging success stories.