Dual gene therapy heals rare lung disease in newborn mice

Our lungs are made up of a thin and delicate layer of cells – so delicate in fact that they would collapse under the pressure of a simple breath of air, were it not for a coating of fatty surfactant. 

Unfortunately, about one in a million babies are born with a genetic disorder that disrupts the production of surfactant. The only cure is a lung transplant. 

Researchers from The Ottawa Hospital, CHEO, the University of Ottawa and the University of Guelph are working together to develop a novel gene therapy for genetic surfactant deficiency. 

Their latest study, published in Molecular Therapy, shows that the approach can improve lung function and structure in a mouse model of surfactant protein C (SP-C) deficiency. This is a particularly challenging form of the disorder, as it involves the production of a toxic form of SP-C in addition to the loss of the normal form of SP-C. 

To address this challenge, the team designed a dual-purpose gene therapy that uses an adeno associated virus (AAV) to produce the effective form of SP-C while also silencing the toxic form. 

“This approach is very promising and we are doing everything we can to move towards clinical trials,” said senior author Dr. Bernard Thébaud, neonatologist and senior scientist at The Ottawa Hospital and CHEO and professor at the University of Ottawa.

Dr. Thébaud also holds a Tier 1 Canada Research Chair in Lung Stem Cell Biology and Regeneration and a uOttawa Partnership Research Chair in Regenerative Medicine and serves as Chief Medical Officer of startup company Inspire Biotherapeutics. 
 

The Ottawa Hospital is a leading academic health, research and learning hospital proudly affiliated with the University of Ottawa and supported by The Ottawa Hospital Foundation. All researchers at The Ottawa Hospital follow a Responsible Innovation Framework for developing and commercializing innovations in a responsible way.