THERAPEUTIC REPAIR OF MUTATED NUCLEIC-ACID SEQUENCES

The principle of therapeutic nucleic acid repair has been demonstrated in cell-free and cell culture experiments, in which compounds bind to and repair mutated sequences, thereby treating the primary defects of genetic disease. The mechanisms used to promote repair are diverse, e ncompassing techniques related to antisense,triple-strand, and ribozym es. Therapeutic nucleic acid repair has the potential to revert mutati ons to wild type, and therefore is more suitable than traditional gene therapy for treating gain-of-function mutations.