Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning

AAV Capsid-Promoter Interactions Determine CNS Cell-Selective Gene Expression In Vivo

(2020) Sara K. Powell et al.   MOLECULAR THERAPY

Multiplexed Cre-dependent selection yields systemic AAVs for targeting distinct brain cell types

(2020) Sripriya Ravindra Kumar et al.   NATURE METHODS

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier

(2019) Juliette Hordeaux et al.   MOLECULAR THERAPY

AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice

(2019) William A. Liguore et al.   MOLECULAR THERAPY

Clade F AAVHSCs cross the blood brain barrier and transduce the central nervous system in addition to peripheral tissues following intravenous administration in nonhuman primates

(2019) Jeff L. Ellsworth et al.   PLoS One

Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids

(2019) Qin Huang et al.   PLoS One

Selection of an Efficient AAV Vector for Robust CNS Transgene Expression

(2019) Killian S. Hanlon et al.   Molecular Therapy-Methods & Clinical Development

Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries

(2019) Pauline F. Schmit et al.   Molecular Therapy-Methods & Clinical Development

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

In Vivo Selection of a Computationally Designed SCHEMA AAV Library Yields a Novel Variant for Infection of Adult Neural Stem Cells in the SVZ

(2018) David S. Ojala et al.   MOLECULAR THERAPY

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain

(2018) Yasunori Matsuzaki et al.   NEUROSCIENCE LETTERS

Helper-free Production of Laboratory Grade AAV and Purification by Iodixanol Density Gradient Centrifugation

(2018) Sean M. Crosson et al.   Molecular Therapy-Methods & Clinical Development

Gene therapy for neurological disorders: progress and prospects

(2018) Benjamin E. Deverman et al.   NATURE REVIEWS DRUG DISCOVERY

Optimization of design and production strategies for novel adeno-associated viral display peptide libraries

(2017) J Körbelin et al.   GENE THERAPY

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

(2017) Ken Y Chan et al.   NATURE NEUROSCIENCE

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

How to Successfully Screen Random Adeno-Associated Virus Display Peptide Libraries In Vivo

(2017) Jakob Körbelin et al.   Human Gene Therapy Methods

A MicroRNA124 Target Sequence Restores Astrocyte Specificity of gfaABC 1 D-Driven Transgene Expression in AAV-Mediated Gene Transfer

(2017) Grit Taschenberger et al.   Molecular Therapy-Nucleic Acids

A brain microvasculature endothelial cell‐specific viral vector with the potential to treat neurovascular and neurological diseases

(2016) Jakob Körbelin et al.   EMBO Molecular Medicine

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

(2016) Michaël Hocquemiller et al.   HUMAN GENE THERAPY

Pulmonary Targeting of Adeno-associated Viral Vectors by Next-generation Sequencing-guided Screening of Random Capsid Displayed Peptide Libraries

(2016) Jakob Körbelin et al.   MOLECULAR THERAPY

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

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

Starcode: sequence clustering based on all-pairs search

(2015) Eduard Zorita et al.   BIOINFORMATICS

A Comprehensive RNA Sequencing Analysis of the Adeno-Associated Virus (AAV) Type 2 Transcriptome Reveals Novel AAV Transcripts, Splice Variants, and Derived Proteins

(2015) Catrin Stutika et al.   JOURNAL OF VIROLOGY

High Capsid–Genome Correlation Facilitates Creation of AAV Libraries for Directed Evolution

(2015) Mathieu Nonnenmacher et al.   MOLECULAR THERAPY

Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna

(2014) Christian Hinderer et al.   Molecular Therapy-Methods & Clinical Development

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

(2013) Leszek Lisowski et al.   NATURE

In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

(2013) D. Dalkara et al.   Science Translational Medicine

Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9

(2012) M. A. DiMattia et al.   JOURNAL OF VIROLOGY

Successful Expansion but Not Complete Restriction of Tropism of Adeno-Associated Virus by In Vivo Biopanning of Random Virus Display Peptide Libraries

(2009) Stefan Michelfelder et al.   PLoS One

Ultrafast and memory-efficient alignment of short DNA sequences to the human genome

(2009) Ben Langmead et al.   GENOME BIOLOGY

GFAPpromoter elements required for region-specific and astrocyte-specific expression

(2008) Youngjin Lee et al.   GLIA

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

(2008) D. Grimm et al.   JOURNAL OF VIROLOGY

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

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY