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.
Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy
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