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Not actual patients.

What is gene therapy?

Gene therapy is changing the way we manage some genetic diseases by targeting missing, nonworking, or faulty genes.


What is gene replacement therapy?

Gene replacement is a type of gene therapy that replaces the function of a missing or nonworking gene with a new, working copy of the malfunctioning gene to treat some genetic conditions.

What research is being done in genetic diseases?

Genetic disease research is making important advancements in our understanding of many genetic diseases and how they can be treated.