Mueller Lab for Gene Therapy

Our latest work is now available online - we describe the first animal model of antitrypsin deficiency.
Alpha-1 Foundation AlphaFriend Maria University of Massachusetts Medical School PNAShttp://www.pnas.org/content/pnas/early/2018/02/15/1713689115.full.pdf

Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema Chronic obstructive pulmonary disease affects 10% of the worldwide population, and the leading genetic cause is a genetic disease, α-1 antitrypsin (AAT) deficiency. Humans have only one gene that codes for the AAT protein, but mice have up to six, which made it impossible for decades to create a mo...

Our latest work is making the November cover of Molecular Therapy! Proof-of-concept treatment for antitrypsin deficiency. Thanks to our collaborators!

"On the Cover: Diastase-resistant periodic acid-Schiff staining of an untreated PiZ mouse liver. The magenta-colored globules are intracellular inclusions of polymers of the Z-form of α-1 antitrypsin, which is associated with liver pathology. As described in this issue by Borel et al. (pp. 2477–2489), treatment with the AAV-GeneRide-DualFunctionAAT vector reduces the amount of globules-positive hepatocytes by more than 100-fold."
Elsevier Alpha-1 Foundation AlphaFriend Maria University of Massachusetts Medical School