Gene Therapy for Cystic Fibrosis
What is Cystic Fibrosis?
Cystic fibrosis is caused by mutations in the gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) channel, causing viscous secretions. Clinically this leads to difficulties breathing and frequent lung infections. The channel has also recently been implicated to function in lymphocytes and may be playing a role in calcium dependent activation of T-cells. It is thought that the dysfunction of the channel in T-cells may have some consequences with predisposes the patients to allergy and reducing their ability to fight lung infections.
Dr. Chris Mueller, PhD
A leading researcher for Cystic Fibrosis
Chris Mueller, PhD, is an Associate Professor of Pediatrics and a faculty member of the Horae Gene Therapy Center. Dr. Mueller received his PhD from the University of Florida and later became an independent investigator at UMass where he established his lab in 2011. Alpha-one antitrypsin has always remained his primary line of research. Dr. Mueller was recently awarded the 2016 Shillelagh Award from the Alpha-1 Foundation, which rewards outstanding Alpha-1 researchers and clinicians.
Dr. Mueller's gene therapy strategies on Cystic Fibrosis
We use gene therapy to express CFTR in the lung or correct the T-cell phenotype by gene therapy or calcium channel modulators.
Other research interests
Currently Dr. Mueller is also developing gene therapy strategies on Alpha-one Antitrypsin (AAT) Deficiency, Amyotrophic Lateral Sclerosis (ALS) and Huntington's Disease.
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Chris Mueller, PhDAssociate Professor, Department of Pediatrics and Horae Gene Therapy Center |
