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References from Engineering novel AAV capsids by broadly attenuated and subsequent muscle-specific tropism in mice and NHPs. Local targets link to admitted publications; unresolved targets remain external evidence.
Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates
10.1016/j.ymthe.2005.08.017 · 2006 · External reference
Adeno-associated virus vector as a platform for gene therapy delivery
10.1038/s41573-019-0012-9 · 2019 · External reference
Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection
10.1038/mt.2008.76 · 2008 · External reference
Adeno-associated virus as a delivery vector for gene therapy of human diseases
2024 · External reference
Minimizing the inhibitory effect of neutralizing antibody for efficient gene expression in the liver with adeno-associated virus 8 vectors
10.1038/mt.2012.258 · 2013 · External reference
Challenges and opportunities of machine-guided capsid engineering for gene therapy
10.18609/cgti.2019.058 · 2019 · External reference
Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV
10.1016/j.ymthe.2020.07.009 · 2020 · External reference
High-dose AAV gene therapy deaths
10.1038/s41587-020-0642-9 · 2020 · External reference
Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne’s Muscular Dystrophy
10.1056/nejmoa2307798 · 2023 · External reference
Lethal immunotoxicity in high-dose systemic AAV therapy
10.1016/j.ymthe.2023.10.015 · 2023 · External reference
AAV-RNAi constructs promote DRG toxicity
10.1016/j.ymthe.2024.12.027 · 2025 · External reference
Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology
10.1089/hum.2020.167 · 2020 · External reference
Breaching the blood-brain barrier: AAV triggers dose-dependent toxicity in the brain
10.1016/j.omtm.2023.09.001 · 2023 · External reference
High-titer AAV disrupts cerebrovascular integrity and induces lymphocyte infiltration in adult mouse brain
10.1016/j.omtm.2023.08.021 · 2023 · External reference
Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy
10.1016/j.ymthe.2018.07.011 · 2018 · External reference
Engineering the AAV capsid to optimize vector–host-interactions
10.1016/j.coph.2015.08.002 · 2015 · External reference
AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset
10.1038/s41593-021-00969-4 · 2022 · External reference
Directed evolution of AAV serotype 5 for increased hepatocyte transduction and retained low humoral seroreactivity
10.1016/j.omtm.2020.10.010 · 2021 · External reference
Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer
10.1007/s40259-023-00585-7 · 2023 · External reference
AAV capsid structure and cell interactions
10.1007/978-1-61779-370-7_3 · 2011 · External reference
Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning
10.1016/j.omtm.2020.12.006 · 2021 · External reference
DELIVERing transgenes into muscle
10.1038/s41592-021-01322-0 · 2021 · External reference
Engineered AAVs for non-invasive gene delivery to rodent and non-human primate nervous systems
10.1016/j.neuron.2022.05.003 · 2022 · External reference
Systemic administration of novel engineered AAV capsids facilitates enhanced transgene expression in the macaque CNS
2023 · External reference
In Vivo Selection of a Computationally Designed SCHEMA AAV Library Yields a Novel Variant for Infection of Adult Neural Stem Cells in the SVZ
10.1016/j.ymthe.2017.09.006 · 2018 · External reference
AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice
10.1016/j.ymthe.2019.07.017 · 2019 · External reference
Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing
10.1038/s41467-022-33745-4 · 2022 · External reference
Characterization of the Relationship of AAV Capsid Domain Swapping to Liver Transduction Efficiency
10.1038/sj.mt.6300293 · 2007 · External reference
Drawing a high-resolution functional map of adeno-associated virus capsid by massively parallel sequencing
10.1038/ncomms4075 · 2014 · External reference
Identification of Amino Acid Residues in the Capsid Proteins of Adeno-Associated Virus Type 2 That Contribute to Heparan Sulfate Proteoglycan Binding
10.1128/jvi.77.12.6995-7006.2003 · 2003 · External reference
Evading and overcoming AAV neutralization in gene therapy
10.1016/j.tibtech.2022.11.006 · 2023 · External reference
Binding and neutralizing anti-AAV antibodies Detection and implications for rAAV-mediated gene therapy
10.1016/j.ymthe.2023.01.010 · 2023 · External reference
Cell Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery
10.1016/j.cell.2021.08.028 · 2021 · External reference
Adeno-Associated Virus Capsid-Promoter Interactions in the Brain Translate from Rat to the Nonhuman Primate
10.1089/hum.2020.196 · 2020 · External reference
Use of adeno-associated virus vector for cardiac gene delivery in large-animal surgical models of heart failure
10.1089/humc.2017.070 · 2017 · External reference
The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice
10.1016/j.ymthe.2018.01.018 · 2018 · External reference
Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids
10.1371/journal.pone.0225206 · 2019 · External reference
Characterization of brain transduction capability of a BBB-penetrant AAV vector in mice, rats and macaques reveals differences in expression profiles
10.1038/s41434-024-00466-w · 2024 · External reference
Ly6a Differential Expression in Blood–Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B
10.1089/hum.2019.186 · 2020 · External reference
Cellular transduction mechanisms of adeno-associated viral vectors
10.1016/j.coviro.2016.08.001 · 2016 · External reference
Cryo-EM Reveals Integrin-Mediated TGF-β Activation without Release from Latent TGF-β
10.1016/j.cell.2019.12.030 · 2020 · External reference
Unresolved reference
External reference
Unresolved reference
2017 · External reference
Satellite cell-specific deletion of Cipc alleviates myopathy in mdx mice
10.1016/j.celrep.2022.110939 · 2022 · External reference
Future of rAAV gene therapy: platform for RNAi, gene editing, and beyond
10.1089/hum.2016.171 · 2017 · External reference
Adeno-associated virus (AAV) capsid engineering in liver-directed gene therapy
10.1080/14712598.2021.1865303 · 2021 · External reference
A comprehensive study of a 29-capsid AAV library in a non-human primate central nervous system
10.1016/j.ymthe.2021.07.010 · 2021 · External reference
A systematic capsid evolution approach performed in vivo for the design of AAV vectors with tailored properties and tropism
10.1073/pnas.1910061116 · 2019 · External reference
A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection
10.1073/pnas.0813207106 · 2009 · External reference
Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy
10.1038/nm.2025 · 2009 · External reference
Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle
10.1038/nbt.1599 · 2010 · External reference
Heparan sulfate proteoglycan binding properties of adeno-associated virus retargeting mutants and consequences for their in vivo tropism
10.1128/jvi.00076-06 · 2006 · External reference
Scratching the surface of RGD-directed AAV capsid engineering
10.1016/j.ymthe.2021.10.020 · 2021 · External reference
Identification of a Heparin-Binding Motif on Adeno-Associated Virus Type 2 Capsids
10.1128/jvi.77.20.11072-11081.2003 · 2003 · External reference
Ancestral library identifies conserved reprogrammable liver motif on AAV capsid
2022 · External reference
Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution
10.1016/j.omtm.2022.12.014 · 2023 · External reference
A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates
10.1016/j.omtm.2018.03.004 · 2018 · External reference
10.1371/journal.ppat.1013533 · 2025 · Admitted local publication
Directed evolution of liver-detargeted AAV vectors for systemic gene delivery to skeletal muscle and heart
2025 · External reference
Long-term follow-up of the first in human intravascular delivery of AAV for gene transfer: AAV2-hFIX16 for severe hemophilia B
10.1016/j.ymthe.2020.06.001 · 2020 · External reference
A rationally engineered capsid variant of AAV9 for systemic CNS-directed and peripheral tissue-detargeted gene delivery in neonates
10.1016/j.omtm.2018.03.004 · 2018 · External reference
Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders
2022 · External reference
An engineered AAV targeting integrin alpha V beta 6 presents improved myotropism across species
10.1038/s41467-024-52002-4 · 2024 · External reference
Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer
10.1016/j.omtm.2018.09.003 · 2018 · External reference
