- Postdoc, Stanford University
- Ph.D., Emory University
- M.S., Southern Illinois University
- BIOL 137
- BIOL 178
The Johnston lab is pursuing the utilization of designer nucleases to cure genetic blood disorders. Nucleases, such as CRISPRs or TALENS, can be designed to produce a cut at a specific location in the genome. These DNA breaks are then repaired by one of two endogenous cellular pathways: 1) nonhomolgous enjoining or 2) homologous recombination. If a nuclease is delivered to a cell in combination with a donor DNA template, the endogenous homologous recombination machinery can repair the break with the donor DNA. In this way, the donor DNA can be designed to facilitate the incorporation of a gene or genes of interest into a specific location in the genome. Therefore, blood disorders that are due to a nonfunctional gene can be corrected with a nuclease and a donor DNA template. Due to the renewal properties of hematopoietic stem cells (HSC), the Johnston lab is currently interested in editing the genome of HSCs as a means to provide a lifelong cure to patients with Hemophilia A.
- Doering, C.B., Denning, G., Dooriss, K., Gangadharan, B., Johnston, J.M., Kerstann K., McCarty, D., Spencer, H.T. (2009) Directed Engineering of a High-Expression Chimeric Transgene as a Strategy for Gene Therapy of Hemophilia A. Mol Ther. 17:1145-1154.
- Johnston, J.M., Doering C.B., Denning G., and Spencer, H.T. (2013) Generation of an optimization of a lentiviral vector encoding a high expression FVIII transgene for the gene therapy of hemophilia A. Gene Ther. 20:607-615
- Johnston, J.M., Denning, G., Moot, R., Whithead, D., Shields, J., Ledoux, J.M., Doering, C.B., Spencer, H.T. (2014) High-throughput screening identifies compounds that enhance lentiviral transduction. Gene Ther. 21:1008-1020.