gene augmentation therapy

gene augmentation therapy

n.
A procedure for correcting metabolic deficiencies caused by a missing or defective gene by having a healthy gene produce the necessary product without actually substituting that gene for the flawed or absent gene in the DNA.
References in periodicals archive ?
Gene augmentation therapy provides a normal copy of a mutated gene into native cells and hence is applicable for the treatment of haploinsufficiency or loss-of-function mutations.
A major milestone in gene augmentation therapy for IRDs was achieved in 2001 using a canine model for LCA2 due to mutations in the gene RPE65 ([RPE65.sup.-/-]).
Advanced stages of retinal degeneration are incompatible with gene augmentation therapy, which, to be successful, requires that the nonfunctional target cells are still alive.