Christian Mueller

Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease.

A. Morton, C. Mueller, D. Howland, 2017, Human gene therapy.

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 .

The pros and cons of immunomodulatory IL-10 gene therapy with recombinant AAV in a Cftr−/−-dependent allergy mouse model

T. Flotte, C. Mueller, M. Campbell-Thompson, 2009, Gene Therapy.

Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques

Allison M. Keeler, Meghan Blackwood, A. Gruntman, 2024, Molecular therapy. Methods & clinical development.