Suzuki, K., et. al. Nature (2016) https://www.ncbi.nlm.nih.gov/pubmed/27851729
In vivo integration of a transgene has historically been the main challenge to advancing gene therapy due to its inefficiency, especially in non-dividing cells. Suzuki et. al. developed a CRISPR/Cas9 system using a novel homology-independent targeted integration strategy to knock-in DNA sequences in both dividing and non-dividing cells. In order to demonstrate the techniques therapeutic potential, Suzuki et. al. used this technique to partially restore retinal function in a retinal degeneration rat model.