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The Goal of Gene Replacement Therapy

 

What is the goal of gene replacement?

When our genes do their jobs, they help produce necessary proteins.

In a person with a monogenic disease, there is one gene or gene pair that is missing or does not work correctly. This can result in the protein being made incorrectly, being made in short supply, or not being made at all.

The goal of gene replacement, a type of gene therapy, is to give the cell a new, working copy of the missing or nonworking gene. This new gene can give the body instructions for making a particular protein the body needs.

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Person without a
monogenic disease

In a person without a monogenic disease, hypothetical Gene 1, Gene 2, and Gene 3 provide instructions to produce the necessary proteins

Person with a
monogenic disease

In a person with a monogenic disease, Gene 1 is missing or malfunctioning and may not be able to produce the necessary proteins

Monogenic disease treated with gene replacement

Gene replacement provides a working copy of Gene 1 to give the body instructions for making the missing protein
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How does gene replacement aim to treat genetic diseases?

Gene replacement uses a new, working gene to treat a genetic disease. This new gene provides the body with instructions for making the particular protein that was missing or in short supply.

Next  Learn about how gene replacement therapy works