Robin J. Ziegler

Detailed Analysis of Structures and Formulations of Cationic Lipids for Efficient Gene Transfer to the Lung

Edward R. Lee, John Marshall, Craig Siegel et al. · Human Gene Therapy · 1996 · 444 citations

Pharmacy, Molecular Biology, Pharmaceutics +17

Glycoengineered Acid α-Glucosidase With Improved Efficacy at Correcting the Metabolic Aberrations and Motor Function Deficits in a Mouse Model of Pompe Disease

Yunxiang Zhu, Ji-Lei Jiang, Nathan Gumlaw et al. · Molecular Therapy · 2009 · 171 citations · Full text

Life Sciences, Biochemistry, Molecular Biology +15

Effective gene therapy in an authentic model of Tay-Sachs-related diseases

María Begoña Cachón-González, Susan Z. Wang, Robin J. Ziegler et al. · Proceedings of the National Academy of Sciences · 2006 · 149 citations · Full text

Life Sciences, Molecular Biology, Neurology +17

Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy

Gregory D Hurlbut, Robin J. Ziegler, Jennifer B. Nietupski et al. · Molecular Therapy · 2010 · 138 citations · Full text

Pharmacy, Molecular Biology, Translational Sciences +13

AAV2 Vector Harboring a Liver-Restricted Promoter Facilitates Sustained Expression of Therapeutic Levels of α-Galactosidase A and the Induction of Immune Tolerance in Fabry Mice

Robin J. Ziegler, Scott Lonning, Donna Armentano et al. · Molecular Therapy · 2003 · 137 citations

Life Sciences, Genetics, Molecular Biology +16