Genetic Disease Research

Because of the promise gene replacement therapy holds in improving the way genetic diseases are treated, many groups of scientists across many disciplines are exploring its role in treating monogenic diseases. That is, genetic diseases where just one gene is missing or does not work right. Many pharmaceutical companies are also taking important steps in developing gene replacement therapy for monogenic diseases.

With each advancement made, gene replacement therapy is changing the way researchers see genetic diseases.

  • A gene under a magnifying glass

    What is a genetic disease?

    Learn more about genetic diseases to better understand gene replacement therapy.

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  • A gene under a microscope

    Which diseases could benefit from the potential of gene replacement therapy?

    Find out which diseases could be forever changed by gene replacement therapy.

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