There are several types of therapies that aim to treat diseases by using genes—and gene replacement therapy is just one type. These approaches replace a nonworking or missing gene, change how your existing genes act, or introduce modified cells into your body.
Let’s take a closer look at the most common types of gene-based therapies.
Check your understanding of gene replacement therapy
How does gene replacement therapy differ from gene editing?
Gene replacement therapy differs from gene editing in that it does not directly change, or edit, a person’s DNA. Instead, a vector delivers a new, working gene into the nucleus of the cell where it can make the protein the body needs. Both methods offer great promise in the treatment of genetic diseases and are the result of decades of research in the laboratory, in animal models, and in clinical trials with humans.Discover the history of gene-based therapies.
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