Efficient CNS targeting in adult mice by intrathecal infusion of single-stranded AAV9-GFP for gene therapy of neurological disorders

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

Amit C. Nathwani, Edward G. D. Tuddenham, Savita Rangarajan et al. · New England Journal of Medicine · 2011 · 1.8K citations · Full text

Medicine, Hematology, Molecular Medicine +13

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

Kevin D. Foust, Emily Nurre, Chrystal L. Montgomery et al. · Nature Biotechnology · 2008 · 1.4K citations · Full text

Medicine, Neurobiology, Neuroscience +8

Cross-Packaging of a Single Adeno-Associated Virus (AAV) Type 2 Vector Genome into Multiple AAV Serotypes Enables Transduction with Broad Specificity

Joseph E. Rabinowitz, Fabienne Rolling, Chengwen Li et al. · Journal of Virology · 2002 · 750 citations · Full text

Life Sciences, Biotechnology, Molecular Biology +14

Human synapsin 1 gene promoter confers highly neuron-specific long-term transgene expression from an adenoviral vector in the adult rat brain depending on the transduced area

Sebastian Kügler, Mathias Bähr · Gene Therapy · 2003 · 422 citations

Medicine, Neurobiology, Neuroscience +16

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

Steven J. Gray, Sahana Nagabhushan Kalburgi, Thomas J. McCown et al. · Gene Therapy · 2013 · 372 citations

Pharmacy, Molecular Pharmaceutics, Neurobiology +14