This website was developed and funded by BioMarin Pharmaceutical, Inc.

Gene Therapy FAQs

Many of the things about us are passed on from one generation to another through genes.

What are genes?

How do genes, DNA, and proteins fit together?

Parts of the Body

What does gene therapy mean?

How is gene therapy designed to work?

New Functional Gene
Packaged in a Vector, or Viral Shell
Delivered to the Body

What are the possible risks and benefits of gene therapies?

Possible risks:

Possible benefits: