Duncan Hall, Room 535
Telephone: (408) 924-4904
FAX: (408) 924-4840
Postdoc, Stanford University
Ph.D., Emory University
M.S., Southern Illinois University
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.