AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset

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

Ken Y. Chan, Min Jee Jang, Bryan B. Yoo et al. · Nature Neuroscience · 2017 · 1.7K citations

Pharmacy, Biotechnology, Neuroscience +16

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

Carmela Zincarelli, Stephen Soltys, Giuseppe Rengo et al. · Molecular Therapy · 2008 · 1.5K citations · Full text

Life Sciences, Biology, Molecular Biology +15

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

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

Benjamin E. Deverman, Piers L. Pravdo, Bryan P. Simpson et al. · Nature Biotechnology · 2016 · 1.1K citations · Full text

Life Sciences, Genetics, Molecular Biology +14

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

Christian Hinderer, Nathan Katz, Elizabeth L. Buza et al. · Human Gene Therapy · 2018 · 796 citations · Full text

Medicine, Neurobiology, Neuroscience +17