Systemic administration of novel engineered AAV capsids facilitates enhanced transgene expression in the macaque CNS

SWISS-MODEL: homology modelling of protein structures and complexes

Andrew Waterhouse, Martino Bertoni, Stefan Bienert et al. · Nucleic Acids Research · 2018 · 13.6K citations · Full text

Life Sciences, Biochemistry, Bioinformatics +15

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell et al. · New England Journal of Medicine · 2017 · 2.3K citations · Full text

Medicine, Genetics, Neurology +16

CellProfiler 4: improvements in speed, utility and usability

David R. Stirling, Madison J. Swain-Bowden, Alice Lucas et al. · BMC Bioinformatics · 2021 · 2.2K citations · Full text

Engineering, Bioinformatics, Cell Biology +12

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