A new gene is injected into an adenovirus vector, which is used to introduce the modified DNA into a human cell. If the treatment is successful, the new gene will make a functional protein.
Gene therapy is an evolving technique used to treat inherited diseases. The medical procedure involves either replacing, manipulating, or supplementing nonfunctional genes with healthy genes.
For example, to reverse disease caused by genetic damage, researchers can isolate normal DNA and package it into a vector – a molecular delivery vehicle usually made from a disabled virus. The vector is then used to infect a target cell, usually from a tissue affected by the illness, such as liver or lung cells. The vector unloads its DNA cargo, which then begins producing the missing protein and restores the cell to normal.