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Gene therapy

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Human genetic manipulation may require both silencing a gene that produces a harmful protein and/or adding a gene to produce a needed one. This involves introducing exogenous DNA into the person’s cells. This DNA may come from another organism or be synthesized in a lab.

The general term for genetic manipulation in humans for medical reasons is gene therapy. Gene therapy can be done in developed organisms rather than germ lines in several ways. One is to physically restrict the injection of the introduced DNA, such as in the eye to treat blindness, so that the gene can’t get into any other body cells. Another way is using a tissue or cell type-specific promoter with the DNA so that it’s only expressed in particular tissues.

Typically, the introduced DNA is packaged within a vector that is used to get the DNA inside cells within the body. One common vector is a virus, because viruses normally function by binding the cell membrane and inserting their DNA or RNA into the cell. The contents of a virus can be modified to produce a human gene. Also, exogenous DNA may be introduced that encodes a therapeutic protein that works like a drug rather than the DNA corresponding to any actual human gene.

Neurobiology For Dummies

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