Immune profiling of adeno-associated virus response identifies B cell-specific targets that enable vector re-administration in mice

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A B cell-deficient mouse by targeted disruption of the membrane exon of the immunoglobulin μ chain gene

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Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems

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Pharmacy, Biotechnology, Neuroscience +16

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

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Life Sciences, Biology, Molecular Biology +15

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Amit Nathwani, Ulreke M. Reiss, Edward G. D. Tuddenham et al. · New England Journal of Medicine · 2014 · 1.3K citations · Full text

Medicine, Genetics, Molecular Genetics +17