Spotlight on Gene Therapy: finding a Cure

We are thrilled that our lab represented by Chris Mueller received the Outstanding Poster Prize at ASGCT 2015 in New Orleans, Louisiana for our poster: "Simultaneous Disruption of Five SerpinA1 Genes in Mice Using CRISPR/Cas9 to Generate the First Animal Model of Alpha-1 Antitrypsin Deficiency".

In this poster, we described how we generated the first AAT knockout mouse model using the CRISPR/Cas9 technology. Up to now, there was no mouse model for alpha-one antitrypsin deficiency, and these mice will be valuable to test new therapeutics.