“Overcoming this obstacle to gene therapy for surfactant deficiency provides life-altering hope to families and opens up possibilities for future clinical trials,” said Dr. Bernard Thébaud.A team led by Drs. Bernard Thébaud, Sarah Wootton and Martin Kang found a way to give multiple doses of gene therapy for a deadly newborn lung disease, dramatically extending the lives of mice with this condition.
The team previously developed a gene therapy for Surfactant Protein B deficiency. They found a single dose increased the lifespan of newborn mice from two days to six months. Unfortunately, these effects faded over time, and additional doses were blocked by the immune system.
To solve this, the team added genetic sequences to the therapy to “hide” it from the immune system. In experiments published in the American Journal of Respiratory Cell and Molecular Biology, they proved this modified therapy could be given multiple times and helped newborn mice survive up to 12 months.
“Overcoming this obstacle to gene therapy for surfactant deficiency provides life-altering hope to families and opens up possibilities for future clinical trials,” said Dr. Bernard Thébaud, neonatologist and senior scientist at The Ottawa Hospital and CHEO and professor at the University of Ottawa.
Authors: Martin H. Kang, Sylvia P Thomas, Caralyn Westley, Thomas Blouin, Liqun Xu, Ying Kai Chan, Erin Lisk, Sarah Allen, Arul Vadivel, Kennedy Nangle, Janani Ramamurthy, Yanlong Pei, Lunndon Lewis, Jessica J Chiang, Marty J Romeo, Silvia Vaena, Elizabeth C O'Quinn, Henry D Schrecker, Casey G Langdon, Paul J Nietert, George M Church, Jeffrey A Whitsett, Sarah K. Wootton, and Bernard Thébaud
Funding and affiliations: National Institutes of Health, American Society of Gene and Cell Therapy, Canadian Institutes of Health Research, Stem Cell Network, Medical University of South Carolina, The Ottawa Hospital, CHEO, University of Ottawa, University of Guelph, Harvard University, Cincinnati Children’s Hospital Medical Center, and the University of Cincinnati College of Medicine.
Core resources: Preclinical Imaging, Translational Science Lab Histology Core, Biorepository & Tissue Analysis Core
