Chris Cunningham, PhD, and Thanos Tzounopoulos, PhD: Developing cell-type-specific gene therapies for hereditary forms of hearing loss.
Fifty to eighty percent of congenital forms of hearing loss are due to DNA mutations. To this point, gene therapies have lacked specificity, leading to inefficient rescue and sometimes side effects. The Cunningham and Tzounopoulos labs have developed a novel, cell-type-specific gene therapy that is highly specific to one cell type in the cochlea. Their therapy is highly effective at restoring hearing function in pre-clinical mouse models of human deafness without causing side effects. Drs Cunningham and Tzounopoulos have received support for this project from the NIH, Eye & Ear Foundation of Pittsburgh, the University of Pittsburgh Innovation Institute, and the PA Lions Hearing Research Foundation. Drs. Cunningham, Tzounopoulos, and Zevallos have co-founded a company, Echogenesis Therapeutics, aimed at translating their findings in mice into clinical trials for human patients.
Chris Cunningham, PhD, and Melissa McGovern, PhD: Developing gene therapy tools to regenerate hair cells.
Many forms of hearing loss, including genetic, noise-induced, and age-related, can cause irreversible death of cochlear hair cells. The Cunningham and McGovern labs are working together to develop gene therapy tools that can be used to convert non-sensory supporting cells in the adult cochlea into hair cells to restore hearing. They have promising data in mice suggesting that their tools can regenerate hair cells in vivo.
Chris Cunningham, PhD, and Thanos Tzounopoulos, PhD: Investigating the impacts of cochlear gene therapy on function of critical brain regions for auditory processing in deaf animals.
Proper hearing involves cochlear function but also critical auditory processing in the brain. Most of the research focused on gene therapeutic hearing restoration has been limited to investigating the impacts on the cochlea, with few insights into how the brain responds to rescue of cochlear function. Drs. Cunningham and Tzounopoulos are using sophisticated imaging in the auditory cortex of awake mice treated with gene therapies to examine sound-evoked activity in rescued mice and how it compares to normal hearing mice.