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Gene replacement uses a new, working copy of the malfunctioning gene to replace the function of a nonworking or missing gene. This new gene is able to give the body instructions for making a particular protein the body needs.

This gene is created in a laboratory and then packaged in a delivery vehicle called a vector. This vector carries the gene into the nucleus of specific cells. Depending on the vector used, the gene may become part of the cell’s DNA, or it may stay separate. Either way, it starts to make the protein that’s missing or in short supply.

Gene therapy has been an area of study in humans since 1990. In late 2017, a gene replacement therapy was approved for the first time in the US to treat a rare, inherited form of vision loss. 

Many gene therapies are currently being investigated in clinical trials.

Illustrations of a missing or nonworking gene, a new working copy of the gene, and of a vector entering the nucleus of a cell