Chemical Science · 2019 · 89 citations · 33 references
PharmacyBiotechnologyMolecular BiologyDrug DeliveryGene DeliveryVirologyGene Delivery SystemsGene Delivery VectorsIn Vivo Gene DeliveryNucleic Acid DeliveryVector DesignAdeno-associated Viral VectorsAdenoviral VectorsAmino FunctionalizationAntibodiesCapsid EngineeringChemical ModificationHost Cell TropismViral Vector Delivery
Bioconjugated AAV vectors, achieved by coupling of ligands on amino groups of the capsid, are of great interest for gene delivery. Chemical modifications can be used to enhance cell tropism and to decrease interactions with neutralizing antibodies.
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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
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Clades of Adeno-Associated Viruses Are Widely Disseminated in Human Tissues
Guangping Gao, Luk H. Vandenberghe, Mauricio R. Alvira et al. · Journal of Virology · 2004 · 1.1K citations · Full text
Christian Hinderer, Nathan Katz, Elizabeth L. Buza et al. · Human Gene Therapy · 2018 · 796 citations · Full text
Medicine, Neurobiology, Neuroscience +17