Welcome to the ElMallah Lab
Targeting respiratory insufficiency in genetic diseases
Our lab focuses on respiratory pathology and control of breathing in genetic mouse models of neuromuscular and respiratory diseases. Our focus is on monogenetic diseases such as Pompe disease and VLCAD deficiency - both are ideal targets for gene therapy. We are also collaborating with others in the study of smooth muscle dysfunction, novel capsids for Pompe disease and respiratory studies in alpha-1-antitrypsin and ALS.
Pompe disease is a fatal neuromuscular disorder resulting from mutations in the gene for acid alpha-glucosidase (GAA) – an enzyme necessary to degrade lysosomal glycogen. Infants with Pompe disease suffer with respiratory insufficiency often leading to mechanical ventilation. Through a series of pre-clinical studies we are focused on directly addressing respiratory problems in Pompe disease using gene therapy.
The Elmallah Lab is part of the Horae Gene Therapy Center (GTC), which is dedicated to developing therapeutic approaches for rare inherited disease for which there is no cure such as Canavan Disease, Tay-Sachs and Sandhoff diseases, Retinitis Pigmentosa, Cystic fibrosis, Lou Gehrig's disease, Huntington's disease, as well as therapies for the more common diseases such as Hypercholesterimia and Cardiac arrhythmia. The GTC faculty utilizes state of the art technologies to either edit mutated genes that produce disease-causing proteins or introduce a healthy copy of a gene if the mutation results in a non-functional protein.
The Elmallah Lab for Gene Therapy Center is always interested in possible partnerships with both indivituals and organizations. Some possible partnerships include knowledge exchange, consulting, collaborations with academia and/or industry, reagent exchange, sponsored research and spinout activities. We encourage you to contact us and learn more how we can partner together to advance the area of gene therapy.
We believe that great scientific discoveries happen when people work together.
Let's have the next big breakthrough together!