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What is Gene Therapy?


What Is Gene Therapy?

The goal of gene therapy is to introduce new genetic information into cells to alter disease phenotypes or kill cells, especially in cancer gene therapy. This is accomplished using viral and non-viral vectors carrying expression cassettes encoding therapeutic or cytotoxic genes, as well as by delivery of small chemically modified oligonucleotides to modulate mRNA metabolism and splicing. Viral vectors are used for direct in vivo delivery of therapeutic genes to specific tissues, or ex vivo to modify patient derived cells that are transplanted back to the same individual at a later time. For more information on Gene Therapy consult the NIH handbook on Gene Therapy.


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