About Gene Replacement Therapy

Gene replacement therapy is one type of gene-based therapy. Explore the links below to learn how this approach attempts to address the root cause of genetic diseases.

  • A new working gene

    What does gene replacement therapy do?

    Gene replacement therapy uses a new, working gene to treat or prevent diseases.

    Learn more about the goal of gene replacement therapy.
  • The science behind gene therapy

    What is the science behind gene replacement therapy?

    Gene replacement therapy aims to replace what the body is missing. Discover the science behind gene replacement therapy.

    Learn how it works.
  • A therapy is made up of a gene and a vector

    What are the key components of gene replacement therapy?

    Gene replacement therapy is made from 2 main components: genes and vectors.

    Learn how it’s made.
  • An icon representing different types of treatment approaches

    Are there different types of gene-based therapies?

    There are several different types of treatment approaches that use genetic materials to treat or prevent genetic diseases. Gene replacement therapy is one type of gene-based therapy.

    Learn more about different types of gene-based therapies.
  • Hourglass

    Are gene-based therapies new?

    Scientists have been investigating for decades how gene-based therapies, like gene replacement therapy, can be used to treat genetic diseases. However, it is only within the past few years that gene-based therapies have been approved for use in people with certain genetic diseases.

    Discover the history of gene-based therapies.
  • Glossary

    Glossary

    Some of the terms used when talking about gene-based therapy can be difficult to understand.

    Use this glossary to look up key words.
  • Answers to questions

    Still have questions?

    Answers to questions about gene replacement therapy and other gene-based therapies.

    Refer to Frequently Asked Questions.