Alpha-1 Antitrypsin Deficiency is a genetic disease characterized by low plasma levels of Alpha-1 Antitrypsin (AAT), an inhibitor that protects the alveolar matrix. Low levels of AAT, lead to emphysema, yet an animal model simulating the lung condition didn’t exist. Recently Mueller et al used CRISPR/Cas9 to create a quintuple knockout of the SERPINA1 gene. Here I prove the mouse model created is a complete knockout (KO). No AAT was found in sera of the KOs and there was no inhibition of neutrophil elastase. Also, KO animals developed emphysema after treatment with LPS (a model that simulates bacterial infection), indicating AAT KO. This will serve as the first AAT-deficient model for the lung and could serve as a model for COPD, allowing for discovery of potential treatments.

• This report represents the work of one or more WPI undergraduate students submitted to the faculty as evidence of completion of a degree requirement. WPI routinely publishes these reports on its website without editorial or peer review.

Creator

• Cardozo, Brynn H

Contributors

• Mueller, Christian

Publisher

• Worcester Polytechnic Institute

Identifier

• E-project-011516-183305

Advisor

• Heilman, Destin

Year

• 2015

Sponsor

• University or Massachusetts Medical School.

Date created

• 2015-01-15

Resource type

• Major Qualifying Project

Major

• Biochemistry

Rights statement

• In Copyright

Last modified

• 2023-09-19