Spotlight on Gene Therapy: finding a Cure

We are happy to announce that our lab represented by postdoctoral associate Florie Borel received an Outstanding Poster Award at ASGCT 2017 in Washington, DC for our poster: "Editing out Five SERPINA1 Paralogs to Create a New Mouse Model of Genetic Emphysema". In this poster, we described how we generated the first AAT knockout mouse model using the CRISPR/Cas9 technology. We further characterized those mice and demonstrated that they spontaneously develop lung emphysema.

Up to now, there was no mouse model for alpha-one antitrypsin deficiency lung disease, and these mice will be valuable to test new therapeutics including gene therapy and genome editing.