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Gene Replacement Therapy

Gene replacement uses a new, working copy of a gene to replace the function of a malfunctioning or missing gene. This new gene can 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 nuclei 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 United States to treat a rare, inherited form of vision loss. Many gene therapies are currently being investigated in clinical trials