Christian Mueller

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α ₁ -Antitrypsin: Interim Results

Terence R. Flotte, Bruce C. Trapnell, Margaret Humphries et al. · Human Gene Therapy · 2011 · 322 citations · Full text

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

Mark Brantly, Jeffrey D. Chulay, Lili Wang et al. · Proceedings of the National Academy of Sciences · 2009 · 311 citations · Full text

Cell Therapy, In Vivo Gene Therapy, Transgene Expression +20

Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System

Hongwei Zhang, Bin Yang, Xin Mu et al. · Molecular Therapy · 2011 · 282 citations · Full text

Human C9ORF72 Hexanucleotide Expansion Reproduces RNA Foci and Dipeptide Repeat Proteins but Not Neurodegeneration in BAC Transgenic Mice

Owen M. Peters, Gabriela Toro Cabrera, Hélène Tran et al. · Neuron · 2015 · 252 citations · Full text

Knockout Mouse, Bac Transgenic Mice, Neurodegenerative Diseases +10

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

Julia F. Alterman, Bruno M.D.C. Godinho, Matthew Hassler et al. · Nature Biotechnology · 2019 · 220 citations

Molecular Neuroscience, Signal Transduction, Medicine +10