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    What is Cystic Fibrosis?

    Cystic Fibrosis is a progressive, genetic disease that causes persistent lung infections, and also affects other organs such as the pancreas, liver and kidneys. The disease is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Cystic Fibrosis is the most common lethal, single-gene disorder affecting Northern Europeans and North Americans.

 

Therapeutic Strategies

 

Gene Therapy for Cystic Fibrosis


Mutations in the CFTR gene cause the production of viscous secretions in the lung, which results in frequent lung infections. The CFTR gene has also recently been implicated in the activation of certain cells of the immune system called T-cells. It is thought that the defective CFTR gene in T-cells may predispose cystic fibrosis patients to allergies and reduce their ability to fight lung infections. Dr. Mueller’s lab is using gene therapy to express CFTR in the lung or correct the T-cell defect.

 

Cystic Fibrosis 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

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.