Cynthia Greer

Small molecule screen employing patient-derived iPS hepatocytes identifies LRRK2 as a novel therapeutic target for Alpha1 Antitrypsin Deficiency

C. Mueller, P. Khoshkenar, S. Blackford, 2021, bioRxiv.

Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency

T. Flotte, M. Kay, C. Mueller, 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice

T. Flotte, C. Mueller, F. Borel, 2022, Molecular therapy. Methods & clinical development.

Survival Advantage of Both Human Hepatocyte Xenografts and Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of alpha-1 Antitrypsin Genome-Edited Hepatocytes for Treatment of alpha-1 Antitrypsin Deficiency Deficiency

T. Flotte, D. Greiner, L. Shultz, 2022 .