Welcome to the Mueller Lab for Gene Therapy!
Many rare genetic disorders are well-characterized, however patients suffering from these disorders often have limited or no treatment options. That’s because these diseases are not interesting for Big Pharma.
As an academic lab mostly funded by the NIH and patients foundations, our goal is to develop gene therapy treatments for three of these disorders: amyotrophic lateral sclerosis, alpha-one antitrypsin deficiency, and cystic fibrosis, using AAV vectors. Learn more about our lab and our programs and check out our publications.
Alpha-one antitrypsin deficiency has been the focus of our lab since its inception. Building up on the work of Dr Terry Flotte directed towards production of the normal AAT protein (see the results of our clinical trial here), we developed a dual function vector that not only allows production of the normal AAT but also silences the mutated, toxic Z-AAT protein.
We are currently looking for funding to take this work - published in 2012 in Molecular Therapy (read here) - further. We already have received the support of The Alpha-1 Project and are looking for one more partner.
In 2012 our lab started a collaboration with Dr Bob Brown, a world leader in the field of ALS. Together we have developed a gene therapy to silence SOD1, the second most prevalent gene linked to ALS.
We recently demonstrated that ALS mice that received this gene therapy survive longer, and that their motor and respiratory functions are maintained. This is particularly promising given that we treated adult mice (as opposed to neonates or juveniles) and that the observed improvements are the best ones ever obtained in adult-treated mice. We also show that this therapy does translate in larger animals, namely in marmoset monkeys.
The Mueller 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.
Partner with Us
Our lab 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 believe that great scientific discoveries happen when people collaborate.
Let's have the next big breakthrough together!