Gene Therapy

In gene therapy, genetic material that provides a missing or necessary protein, or causes a clinically-relevant biochemical process, is introduced into an organ for a therapeutic effect. For gene-based therapies (specifically, those using DNA sequences), it is critical that the desired gene be introduced into organ stem cells in order to achieve long-term expression and therapeutic effect. Although techniques for delivering the therapeutic DNA have been greatly improved since the first gene therapy protocol almost 10 years ago, there are as yet no bona fide successes. Besides delivery problems, loss of expression or insufficient expression is an important limiting factor in successful application of gene therapy and could be overcome by transferring genes into stem cells (which presumably will then differentiate and target correctly).

Email link | Printer-friendly | Feedback | Contributed by: AAAS DoSER and the Institute for Civil Society