Ribozyme Therapy

A form of gene therapy that reduces the amount of "bad" gene product ("bad" RNA) to allow "good" protein to be made from good, non-mutated RNA.

Ribozyme therapy is a form of gene therapy normally used for autosomal dominant forms of retinal degeneration (to reduce the amount of "bad" gene product in a cell). In autosomal dominant disease, individuals have one healthy functioning gene and one gene with a disease-causing mutation. The mutant gene produces a dysfunctional protein that damages the photoreceptor cell. In this instance, ribozymes disrupt the mutant gene's ability to produce the harmful protein by "chewing-up" the gene's RNA messengers. For autosomal recessive disease, ribozyme therapy can be used in combination with gene therapy to install a "good" copy of the gene that produces RNA that is resistant to the ribozyme.