Dr. Christian Mueller, Associate Professor in the Department of Pediatrics and in the Horae Gene Therapy Center at the University of Massachusetts Medical School will give a presentation on the basics of gene therapy at the 24th Annual HPS Network Conference, March 12 at the Long Island Marriott in Uniondale, N.Y. While there currently is not a gene therapy or gene therapy clinical trial for Hermansky-Pudlak Syndrome, scientists are working on potential gene therapies in the lab. Dr. Mueller will help educate the HPS community about what gene therapy really is, how it works and how it might one day benefit patients with Hermansky-Pudlak Syndrome.

Dr. Mueller holds a PhD in Genetics from the University of Florida. He subsequently completed his postdoctoral work as a Parker B Francis Fellow while working on his Master in Clinical Investigation at the University of Massachusetts Medical School. In 2011 he opened the Mueller Lab for Gene Therapy in the Horae Gene Therapy Center. The main focus of his lab is recombinant adeno-associated virus (rAAV)-mediated gene therapy for rare genetic diseases such as alpha-one antitrypsin deficiency (AATD), amyotrophic lateral sclerosis (ALS), and Huntington’s disease (HD). Dr. Mueller is also investigating adaptive immune responses to AAV capsid and is currently collaborating on various AAV clinical trials to characterize these immune responses in patients. One of these collaborations has resulted in the appreciation that human patients treated with intra-muscular injections of rAAV1 can elicit capsid specific T-regulatory immune responses to the capsid. Dr Mueller also pioneered the use of rAAV to deliver artificial miRNAs to silence genes with toxic gain-of-function products. Some of this research culminated with the development of a dual function rAAV vector that is able to augment normal alpha-one antitrypsin (AAT) while simultaneously silencing mutant AAT, as well as rAAV vectors for silencing SOD1, C9ORF72 and HTT as therapies for ALS and HD. Dr Mueller is currently translating the dual-function vector for AATD as well as those for ALS into the clinic.

“To me art and science go hand in hand, as a young artist I enjoyed exploring the boundaries of my imagination, using the tools and techniques I had learned to explore new ways of expressing my thoughts and interpreting the world around me,” said Dr. Mueller, “Although art was my earliest motivator my aspirations to be an artist got re-focused when as a young adult I faced a life-threatening disease. I was grateful to have been cured thanks to recent advances in medical research at the time. I realized that the opportunity that science had bestowed on me through its advances in medicine was to be my new muse. Nowadays that creativity and curiosity is played out on the lab with the hope that one day the lab and its members can be part of collective that helps heal another person so they too can realize their dreams.” 

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