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Therapeutic Strategies


Gene Therapy to Augment Serum AAT levels

The labs of Drs. Flotte and Mueller aim to boost AAT levels in Alpha-1 antitrypsin deficiency patients using gene therapy approaches involving adeno-associated virus (AAV) vector-mediated delivery of AAT. The main advantage of gene therapy over recombinant protein augmentation therapy is that it would be a one-time treatment, as opposed to weekly or biweekly infusions.


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Alpha-1 Antitrypsin Deficiency Investigators

terence flotte md

Terence Flotte, MD

Professor of pediatrics, Celia and Isaac Haidak Professor of Medical Education

Flotte Lab

chris mueller phd

Chris Mueller, PhD

Associate Professor

Mueller Lab

Erik Sontheimer, PhD

Erik Sontheimer, PhD

Professor, RNA Therapeutics Institute

Sontheimer Lab

Clinical Trials

At the Li Weibo Institute for Rare Diseases Research, our researchers and clinicians are dedicated to finding new treatment options for patients. Our clinical trials program is another way we continue to advance treatments and make new discoveries. If you or someone you know may be interested in a clinical trial, please subscribe for updates and you will be notified when we open up a new trial.


More About Our Research on Alpha-1 Antitrypsin Deficiency