Table 1.
Subsidiary categories of gene therapy.
Type of Gene Therapy | Definition | Example |
---|---|---|
Direct | The intentional modification of a specific somatic gene in order to correct defective genes or to fix an allele's malign function. | Single-base pair editing to resolve insertions or deletions that lead to defective proteins. |
Compensatory | The induction of expression of genetic material that compensates for lost or aberrant cellular function. | Editing of gene to increase or decrease expression in target cell, or induce expression normally turned off. |
Augmenting | The introduction of a novel function that would not otherwise be present in the target cell type. | Editing of a gene to provide a different function from what is typical; insertion of a new version of a gene into the genome (as opposed to editing the existing version) |