Stem cells are ideal for gene therapy because they appear to be immortal. Cells carrying corrective genes are introduced directly into the tissue where the genes are needed. This treatment is good for conditions that are localized, but it cannot correct systemic disorders. In situ treatment is being explored for diseases like cystic fibrosis, muscular dystrophy, and cancer. This therapy does not exist yet but is the therapy of the future.
Researchers hope to inject gene carriers into the bloodstream.
Once in the body the carrier genes will find their target cells, deliver their genetic information efficiently and safely while ignoring other cell types. Progress has been made developing toward vectors that will home to specific cell types. Scientists have developed several methods for transporting genetic material for ex vivo therapy. The most effective therapy uses modified viruses as carriers. Viruses are useful because they are able to penetrate cells and insert the genetic material that they contain into the new host.