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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).
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| Contributed by: AAAS DoSER and the Institute for
Civil Society
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