A gene therapy in development by researchers at The Ottawa Hospital, CHEO and uOttawa in collaboration with the University of Guelph, is showing dramatic results in mice with this disorder, improving their life expectancy from two days to over six months, according to a study published in Nature Communications.
This gene therapy may also be able to treat other debilitating and life-threatening genetic lung disorders that appear later in children and young adults. Led by Dr. Thébaud and Dr. Wootton, the team recently received $364,000 from the Stem Cell Network to pursue large-scale manufacturing of AAV and to take the steps necessary to make it ready for clinical trials.