Gene Therapy (GT)

A therapeutic process that replaces or turns off the "bad" or mutated disease-causing gene and restores some level of normal protein function.

Gene therapy (GT) is one of the Foundation's seven research priority areas and can be defined as the delivery of a gene or genetic information into cells for the purpose of achieving a therapeutic effect. At least three forms of gene therapy (gene replacement therapy, ribozyme therapy and RNA interference or RNAi) have shown promise in the treatment of retinal degenerative diseases. Gene replacement therapy is replacing the "bad" gene with the "good" form; Ribozyme therapy chews-up the "bad" RNA (the gene messenger) so that it can't make a "bad" protein that will do harm; and RNAi treatment causes the "bad" RNA to be destroyed by existing cell defense processes.