Spotlight on Gene Therapy: finding a Cure

We are happy to announce that our annual Alpha-1 Awareness Day will be held on November 17th at UMass!

What is alpha-one antitrypsin deficiency?  

Alpha-one antitrypsin (AAT) deficiency is a common (although often unrecognized) genetic disease, with up to 4% of the population carrying an abnormal AAT gene. Patients can suffer from either a lung disease, which is caused by a loss of the normal AAT action, or from a liver disease, which is caused by build-up of the abnormal mutant protein (called Z-AAT) within the liver cells.

AAT deficiency occurs worldwide, but the prevalence is variable depending on regions. It is a rare disease in Europe and in the US. It affects between 1:1500 and 1:3500 in people of European descent. It is the 2nd most common genetic disorder in Ireland, where 1:25 people carry the mutant gene. 

About our event on November 17th   

We will host a special day-long event at UMass together with the Alpha-1 Foundation. The goal is to raise awareness for alpha-one antitrypsin deficiency within the UMass community.

Visitors can learn more about Alpha-1 and its symptoms, but also about genetic testing and treatment. It will also be an occasion to meet Alpha-1 patients (called Alphas), and hear their story. They will be several Alphas and UMass researchers present on each stand who will be able to inform visitors and answer any questions.

You are invited to come see our stands in the lobby of the Albert Sherman building and in the hospital lobby. Wear purple when you visit our stands and we'll snap a pic and post it on our website!

You can also see last's year stand here.

Our approach to find a cure for Alpha-1 Deficiency: Developing Gene Therapy strategies  

Our Lab has developed a novel, proprietary gene therapy platform and applied it to develop a gene therapy treatment for alpha-one antitrypsin deficiency. Read more