Genome Engineering and Gene Therapy

Genome Engineering

The Wert laboratory uses gene-editing techniques (e.g. CRISPR) to introduce human patient mutations into stem cells and mice to model human retinal dystrophies. These techniques are also used to create reporter cell lines to investigate the pathophysiology of disease and follow the cells after transplantation.

  • Bakthavatchalu V*, Wert KJ*, Feng Y, Mannion A, Ge Z, Garcia A, Caron T, Scott K, Madden CM, Jacobsen JT, Victora G, Jaenisch R, Fox JG. (2018) Cytotoxic Escherichia coli strains encoding colibactin isolated from immunocompromised mice with urosepsis and meningitis. PLoS One 13: e0194443. *These authors contributed equally to this work.

Gene Therapy

A key interest of the Wert laboratory is to use models of retinal degenerative disease to test potential therapies for patients suffering from these conditions. One approach is gene therapy, where viral delivery of a normal gene (gene supplementation) or delivery of gene-editing machinery can be delivered into the eye. These gene therapy approaches tested in the laboratory provide preclinical efficacy analysis and can lead to the initiation of future clinical trials for patients.

  • Wert KJ, Davis RJ, Sancho-Pelluz J, Nishina PM, Tsang SH. (2013) Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa. Hum. Mol. Genet. 22:3:558-567.
  • Wert KJ, Sancho-Pelluz J, Tsang SH. (2014) Mid-stage intervention achieves similar efficacy as conventional early-stage treatment using gene therapy in a pre-clinical model of retinitis pigmentosa. Hum. Mol. Genet. 23:2:514-523.
  • Wert KJ, Skeie JM, Bassuk AG, Olivier AK, Tsang SH, Mahajan VB. (2014) Functional validation of a human CAPN5 exome variant by lentiviral transduction into mouse retina. Hum. Mol. Genet. 23:10:2665-2677.