Experimental Models of Alpha-1 Antitrypsin Deficiency Lung and Liver Disease
The Alpha-1 Foundation joined with researchers, clinicians and industry partners in Bethesda, MD for the 27th Gordon L. Snider (GLS) Critical Issues Workshop titled Experimental Models of Alpha-1 Antitrypsin Deficiency Lung and Liver Disease. The goal of GLS topical workshops, is to provide new information that speeds the journey to a cure for Alpha-1 Antitrypsin Deficiency (AATD). The overall goal of this workshop is to explore experimental models of liver and lung disease and how these models can be applied to both advance understanding of AATD disease mechanisms and assess efficacy of potential therapeutics.
This GLS workshop surveys the current landscape of experimental AATD modeling, touching on models long utilized for this purpose as well as emergent models that have not yet been applied towards this goal. Because each individual model by definition has relative strengths and weaknesses, one focus will be to understand how best to apply each and how complementary approaches might be collectively applies to address existing knowledge gaps.
The GLS Critical Issues Workshop Series facilitates expert exchanges and answers important questions to stimulate additional research. These conferences stand as a cornerstone of A1F’s efforts, igniting enthusiasm within the field and solidifying its reputation as a leader within the rare disease community.
The workshop was divided into two sections, “The Liver” moderated by Dr. Jeffrey Teckman and “The Lung” moderated by Dr. Andrew Wilson.
Thank you to presenters:
- David Rudnick, MD, PhD (Washington University School of Medicine)
- Adam Syanda (Imperial College London)
- Kentaro Iwasawa, MD (Cincinnati Children’s Hospital)
- Amy Stoddard, PhD (MIT, Boston University)
- Leo van Grunsven, PhD (Vrije Universiteit Brussel)
- Foad Rouhani, MD, PHD (Francis Crick Institute)
- Chris Mueller, PhD (Sanofi)
- S. Vamsee Raju, PhD (University of Alabama at Birmingham)
- Carly Merritt, BA (Boston University School of Medicine)
- Melanie Koenigshoff, MD, PhD (University of Pittsburgh School of Medicine)
- Francesca Polverino, MD, PhD (Baylor College of Medicine)
A1F thanks the workshop’s co-chairs Dr. Andrew Wilson and Dr. Tamir Rashid. Thank you to our sponsors for their ongoing commitment to the advancement of research: AlphaNet, Beam Therapeutics, CSL Behring, Grifols and Takeda.
