Publication: Molecular Therapy. 21(6): 1151-1159.
Published Date: June 2013
Examined efficacy of TALEN technology to correct gene mutations and provided a safety profile for translational application of TALEN gene-editing.
Patients with recessive dystrophic epidermolysis bullosa (RDEM) have genetic defects in COL7A1, which the authors sought to correct by capitalizing on endogenous homologous recombination machinery supplemented with TALEN technology. Using primary, patient-derived fibroblasts, the authors used TALENs in a genome-editing strategy from which gene-corrected clones were isolated. Fibroblast clones were expanded and differentiated into inducible pluripotent stem (iPS) cells, which could form skin-like structures in vivo. Additionally, deep sequencing and gene tagging methodology established an unbiased genome-wide screen that identified TALENs as a safe, potential technology in genomic-based medicine.