The genomes of these cells can be relatively easily and precisely manipulated through a technique known as targeted gene replacement. (scientificamerican.com)
«Encouraging data for gene replacement therapy for SMA type I, phase 1 study shows.» (sciencedaily.com)
Indeed, Leber Congenital Amaurosis (LCA) was one of the first recessive disorders to undergo clinical trials for gene replacement therapy. (massgenomics.org)