What is Gene Therapy? – Dana-Farber/Boston Children’s …

Gene therapy is a technique throughwhich genes are added or replaced to treat or prevent disease.

Our genes, which hold the code for all of our body's functions, aremade of DNA. Damage to DNA, such as a mutation, is an underlying cause of thegenetic defects that lead to cancers, blood disorders, and other conditions.Gene therapy delivers DNA into a patients cells to replace faulty or missinggenes or add new genes in an attempt to cure cancer or make changes so thebody is better able to fight off disease.

Scientists are investigating a number of different ways to do this:

How does gene therapy deliver new genes into cells?

With gene therapy, the DNA for the new or corrected gene or genes iscarried into a patients cells by a delivery vehicle called a vector, typicallya specially engineered virus. The vector then inserts the gene(s) into thecells' DNA.

For patients, the process for delivering genes to cells is fairlysimple.

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...or click to see an image of the gene therapy process:

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What is Gene Therapy? - Dana-Farber/Boston Children's ...