Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disorder that affects the upper and lower motor neurons resulting in muscle atrophy, paralysis, respiratory failure and death. 5-10% of ALS is familial and 20% of familial ALS overexpresses mutant SOD1. The ALS mouse model (the SOD1G93A mouse) overexpresses mutant SOD1 and recapitulates ALS pathophysiology making it an ideal model to study novel therapies for this disease. Using an adeno-associated viral vector (AAV) carrying a microRNA to knock down SOD1 (AAVrh10-H1-miRSOD1) we sought to correct the respiratory pathology in SOD1G93A mice with the goal of improving respiratory function and overall survival. A one time injection given at 60 days was able to extend survival of ALS mice and improve respiratory function.

• 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

• Semple, Victoria Anne

Colaboradores

• ElMallah, Mai

Publisher

• Worcester Polytechnic Institute

Identifier

• E-project-042617-224920

Advisor

• Dempski, Robert E.

Year

• 2017

Sponsor

• University of Massachusetts Medical School

Date created

• 2017-04-26

Resource type

• Major Qualifying Project

Major

• Biochemistry

Rights statement

• In Copyright

Última modificação

• 2023-09-27