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Spotlight on Gene Therapy: finding a Cure

Outstanding Poster Prize at 2017 ASGCT

Posted On: Wednesday, June 07, 2017 Posted By: Florie Borel Tags: Alpha-one antitrypsin, Presentations

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.