A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates

A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates

Adeno-associated virus (AAV) has provided the gene therapy field with the most powerful in vivo gene delivery vector to realize safe, efficacious, and sustainable therapeutic gene expression. Because many clinically relevant properties of AAV-based vectors are governed by the capsid, much research e...

Full description

Bibliographic Details
Main Authors:	Dan Wang, Shaoyong Li, Dominic J. Gessler, Jun Xie, Li Zhong, Jia Li, Karen Tran, Kim Van Vliet, Lingzhi Ren, Qin Su, Ran He, Jason E. Goetzmann, Terence R. Flotte, Mavis Agbandje-McKenna, Guangping Gao
Format:	Article
Language:	English
Published:	Elsevier 2018-06-01
Series:	Molecular Therapy: Methods & Clinical Development
Online Access:	http://www.sciencedirect.com/science/article/pii/S2329050118300330

Similar Items

Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS
by: Nicholas M. Kanaan, et al.
Published: (2017-09-01)

Completion of the AAV Structural Atlas: Serotype Capsid Structures Reveals Clade-Specific Features
by: Mario Mietzsch, et al.
Published: (2021-01-01)

Mapping the AAV capsid host antibody response towards the development of second generation gene delivery vectors
by: Yu-Shan eTseng, et al.
Published: (2014-01-01)

Sub-2 Å Ewald curvature corrected structure of an AAV2 capsid variant
by: Yong Zi Tan, et al.
Published: (2018-09-01)

Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: the final threshold?
by: George V Aslanidi, et al.
Published: (2013-01-01)

Creating an arsenal of Adeno-associated virus (AAV) gene delivery stealth vehicles.
by: J Kennon Smith, et al.
Published: (2018-05-01)

Site-Directed Mutagenesis Improves the Transduction Efficiency of Capsid Library-Derived Recombinant AAV Vectors
by: Gai Ran, et al.
Published: (2020-06-01)

Adeno-Associated Virus (AAV) Capsid Stability and Liposome Remodeling During Endo/Lysosomal pH Trafficking
by: Bridget Lins-Austin, et al.
Published: (2020-06-01)

Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors.
by: Christine N Kay, et al.
Published: (2013-01-01)

Correction: Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors
by: Christine N. Kay, et al.
Published: (2013-01-01)

Correction: Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors.
by: Christine N. Kay, et al.
Published: (2013-01-01)

Thermal Stability as a Determinant of AAV Serotype Identity
by: Antonette Bennett, et al.
Published: (2017-09-01)

Characterization of naturally-occurring humoral immunity to AAV in sheep.
by: Joseph Tellez, et al.
Published: (2013-01-01)

Characterization of AAV-Specific Affinity Ligands: Consequences for Vector Purification and Development Strategies
by: Mario Mietzsch, et al.
Published: (2020-12-01)

Adeno-associated virus capsid assembly is divergent and stochastic
by: Tobias P. Wörner, et al.
Published: (2021-03-01)

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes
by: Daniel J Hui, et al.
Published: (2015-01-01)

Optimizing the Targeting of Mouse Parvovirus 1 to Murine Melanoma Selects for Recombinant Genomes and Novel Mutations in the Viral Capsid Gene
by: Matthew Marr, et al.
Published: (2018-01-01)

Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors
by: Hildegard Büning, et al.
Published: (2019-03-01)

Comparison of Liver Detargeting Strategies for Systemic Therapy with Oncolytic Adenovirus Serotype 5
by: Tien V. Nguyen, et al.
Published: (2017-08-01)

A Comparative Analysis of the Structural Architecture of ssDNA Viruses
by: Antonette Bennett, et al.
Published: (2008-01-01)

Structural Characterization of Cuta- and Tusavirus: Insight into Protoparvoviruses Capsid Morphology
by: Mario Mietzsch, et al.
Published: (2020-06-01)

Microglia-specific targeting by novel capsid-modified AAV6 vectors
by: Awilda M Rosario, et al.
Published: (2016-01-01)

Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders
by: Marina Pavlou, et al.
Published: (2021-04-01)

Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries
by: Pauline F. Schmit, et al.
Published: (2020-06-01)

Site-Specific N-Glycosylation on the AAV8 Capsid Protein
by: Arya Aloor, et al.
Published: (2018-11-01)

Random Insertion of mCherry Into VP3 Domain of Adeno-associated Virus Yields Fluorescent Capsids With no Loss of Infectivity
by: Justin Judd, et al.
Published: (2012-01-01)

CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector
by: Giridhar Murlidharan, et al.
Published: (2016-01-01)

AAV-S: A versatile capsid variant for transduction of mouse and primate inner ear
by: Maryna V. Ivanchenko, et al.
Published: (2021-06-01)

Characterization of the GBoV1 Capsid and Its Antibody Interactions
by: Jennifer Chun Yu, et al.
Published: (2021-02-01)

MicroRNA199a-Based Post-transcriptional Detargeting of Gene Vectors for Hepatocellular Carcinoma
by: Bijay Dhungel, et al.
Published: (2018-12-01)

Selection of an Efficient AAV Vector for Robust CNS Transgene Expression
by: Killian S. Hanlon, et al.
Published: (2019-12-01)

Detargeting Lentiviral-Mediated CFTR Expression in Airway Basal Cells Using miR-106b
by: Soon H. Choi, et al.
Published: (2020-10-01)

Tyrosine Mutation in AAV9 Capsid Improves Gene Transfer to the Mouse Lung
by: Sabrina V. Martini, et al.
Published: (2016-07-01)

Capsid serotype and timing of injection determines AAV transduction in the neonatal mice brain.
by: Paramita Chakrabarty, et al.
Published: (2013-01-01)

Characterisation of AAV capsid-specific CD8+ T cell responses in human subjects
by: Vandamme, Céline
Published: (2016)

Peptide ligands incorporated into the threefold spike capsid domain to re-direct gene transduction of AAV8 and AAV9 in vivo.
by: Stefan Michelfelder, et al.
Published: (2011-01-01)

Age-dependent rAAV Mediated Reconstitution of hASPA Reveals N-acetylaspartate Regulates Fuel Selection in the Central Nervous System
by: Gessler, Dominic J.
Published: (2020)

Differential effects of two MRI contrast agents on the integrity and distribution of rAAV2 and rAAV5 in the rat striatum
by: Sue Osting, et al.
Published: (2014-01-01)

Mining the Protein Data Bank to Differentiate Error from Structural Variation in Clustered Static Structures: An Examination of HIV Protease
by: Robert McKenna, et al.
Published: (2012-03-01)

Enhanced Transduction of Human Hematopoietic Stem Cells by AAV6 Vectors: Implications in Gene Therapy and Genome Editing
by: Hua Yang, et al.
Published: (2020-06-01)

Cannot write session to /tmp/vufind_sessions/sess_1omftk1qje20p5gqt7iom9ttls