Therapeutic repair of mutated nucleic acid sequences.

Abstract

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, encompassing techniques related to antisense, triple-strand, and ribozymes. Therapeutic nucleic acid repair has the potential to revert mutations to wild type, and therefore is more suitable than traditional gene therapy for treating gain-of-function mutations.