Systemic AAV vectors for widespread and targeted gene delivery in rodents

Hydrogel-Tissue Chemistry: Principles and Applications

(2018) Viviana Gradinaru et al.   Annual Review of Biophysics

Viral Strategies for Targeting the Central and Peripheral Nervous Systems

(2018) Claire N. Bedbrook et al.   Annual Review of Neuroscience

The Neuropeptide Tac2 Controls a Distributed Brain State Induced by Chronic Social Isolation Stress

(2018) Moriel Zelikowsky et al.   CELL

Dopaminergic dysfunction in neurodevelopmental disorders: recent advances and synergistic technologies to aid basic research

(2018) J Elliott Robinson et al.   CURRENT OPINION IN NEUROBIOLOGY

Widespread transduction of astrocytes and neurons in the mouse central nervous system after systemic delivery of a self-complementary AAV-PHP.B vector

(2018) Melvin Y. Rincon et al.   GENE THERAPY

More expansive gene transfer to the rat CNS: AAV PHP.EB vector dose–response and comparison to AAV PHP.B

(2018) Robert D. Dayton et al.   GENE THERAPY

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

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Cas9/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9.PHP.B-based delivery

(2017) Serena G Giannelli et al.   HUMAN MOLECULAR GENETICS

AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy

(2017) Giuseppe Morabito et al.   MOLECULAR THERAPY

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

(2017) Ken Y Chan et al.   NATURE NEUROSCIENCE

Causal evidence for retina-dependent and -independent visual motion computations in mouse cortex

(2017) Daniel Hillier et al.   NATURE NEUROSCIENCE

Global Representations of Goal-Directed Behavior in Distinct Cell Types of Mouse Neocortex

(2017) William E. Allen et al.   NEURON

Overriding sleep

(2017) Viviana Gradinaru   SCIENCE

Sensory Neurons that Detect Stretch and Nutrients in the Digestive System

(2016) Erika K. Williams et al.   CELL

Extracting structural and functional features of widely distributed biological circuits with single cell resolution via tissue clearing and delivery vectors

(2016) Jennifer Brooke Treweek et al.   CURRENT OPINION IN BIOTECHNOLOGY

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

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

(2016) Yang Yang et al.   NATURE BIOTECHNOLOGY

Visualization of cortical, subcortical and deep brain neural circuit dynamics during naturalistic mammalian behavior with head-mounted microscopes and chronically implanted lenses

(2016) Shanna L Resendez et al.   Nature Protocols

A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons

(2016) D. Gowanlock R. Tervo et al.   NEURON

Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids

(2016) Kyle Chamberlain et al.   Human Gene Therapy Methods

rAAV-compatible MiniPromoters for restricted expression in the brain and eye

(2016) Charles N. de Leeuw et al.   Molecular Brain

Sensitive red protein calcium indicators for imaging neural activity

(2016) Hod Dana et al.   eLife

Comparative next-generation sequencing of adeno-associated virus inverted terminal repeats

(2016) Karl Petri et al.   BIOTECHNIQUES

Vagal Sensory Neuron Subtypes that Differentially Control Breathing

(2015) Rui B. Chang et al.   CELL

Clarifying Tissue Clearing

(2015) Douglas S. Richardson et al.   CELL

Intact-Brain Analyses Reveal Distinct Information Carried by SNc Dopamine Subcircuits

(2015) Talia N. Lerner et al.   CELL

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

ScaleS: an optical clearing palette for biological imaging

(2015) Hiroshi Hama et al.   NATURE NEUROSCIENCE

Whole-body tissue stabilization and selective extractions via tissue-hydrogel hybrids for high-resolution intact circuit mapping and phenotyping

(2015) Jennifer B Treweek et al.   Nature Protocols

Labeling of active neural circuits in vivo with designed calcium integrators

(2015) B. F. Fosque et al.   SCIENCE

Brain BLAQ: Post-hoc thick-section histochemistry for localizing optogenetic constructs in neurons and their distal terminals

(2015) David A. Kupferschmidt et al.   Frontiers in Neuroanatomy

Comparative Analysis of Cesium Chloride- and Iodixanol-Based Purification of Recombinant Adeno-Associated Viral Vectors for Preclinical Applications

(2015) Benjamin Strobel et al.   Human Gene Therapy Methods

CRISPR/Cas9-mediated genome engineering: An adeno-associated viral (AAV) vector toolbox

(2014) Elena Senís et al.   Biotechnology Journal

Single-Cell Phenotyping within Transparent Intact Tissue through Whole-Body Clearing

(2014) Bin Yang et al.   CELL

Engineering adeno-associated viruses for clinical gene therapy

(2014) Melissa A. Kotterman et al.   NATURE REVIEWS GENETICS

Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons

(2014) Jun-Hyeok Choi et al.   Molecular Brain

Empty virions in AAV8 vector preparations reduce transduction efficiency and may cause total viral particle dose-limiting side effects

(2014) Kai Gao et al.   Molecular Therapy-Methods & Clinical Development

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

(2013) F. Mingozzi et al.   BLOOD

A Novel Experimental Strategy to Assess the Metabolic Effects of Selective Activation of a Gq-Coupled Receptor in Hepatocytes In Vivo

(2013) Jian Hua Li et al.   ENDOCRINOLOGY

Comprehensive Expression Analyses of Neural Cell-Type-Specific miRNAs Identify New Determinants of the Specification and Maintenance of Neuronal Phenotypes

(2013) A. Jovicic et al.   JOURNAL OF NEUROSCIENCE

AAV2 production with optimized N/P ratio and PEI-mediated transfection results in low toxicity and high titer for in vitro and in vivo applications

(2013) Xinping Huang et al.   JOURNAL OF VIROLOGICAL METHODS

Mouse Gender Influences Brain Transduction by Intravascularly Administered AAV9

(2013) Casey A Maguire et al.   MOLECULAR THERAPY

Improved tools for the Brainbow toolbox

(2013) Dawen Cai et al.   NATURE METHODS

Capsid Serotype and Timing of Injection Determines AAV Transduction in the Neonatal Mice Brain

(2013) Paramita Chakrabarty et al.   PLoS One

AAV-based shRNA silencing of NF-κB ameliorates muscle pathologies in mdx mice

(2012) Q Yang et al.   GENE THERAPY

Whole Animal Perfusion Fixation for Rodents

(2012) Gregory J. Gage et al.   Jove-Journal of Visualized Experiments

MicroRNA therapeutics

(2011) J A Broderick et al.   GENE THERAPY

Optimizing Promoters for Recombinant Adeno-Associated Virus-Mediated Gene Expression in the Peripheral and Central Nervous System Using Self-Complementary Vectors

(2011) Steven J. Gray et al.   HUMAN GENE THERAPY

Retro-orbital injections in mice

(2011) Tal Yardeni et al.   LAB ANIMAL

Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates

(2011) Steven J Gray et al.   MOLECULAR THERAPY

mGRASP enables mapping mammalian synaptic connectivity with light microscopy

(2011) Jinhyun Kim et al.   NATURE METHODS

Recurrent Circuitry Dynamically Shapes the Activation of Piriform Cortex

(2011) Kevin M. Franks et al.   NEURON

Rapid, Simple, and Versatile Manufacturing of Recombinant Adeno-Associated Viral Vectors at Scale

(2010) Martin Lock et al.   HUMAN GENE THERAPY

MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression

(2010) Jun Xie et al.   MOLECULAR THERAPY

Optogenetic control of heart muscle in vitro and in vivo

(2010) Tobias Bruegmann et al.   NATURE METHODS

The fluorescent protein palette: tools for cellular imaging

(2009) Richard N. Day et al.   CHEMICAL SOCIETY REVIEWS

High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

(2009) E Ayuso et al.   GENE THERAPY

Progress and prospects: immune responses to viral vectors

(2009) S Nayak et al.   GENE THERAPY

Transient Transfection Methods for Clinical Adeno-Associated Viral Vector Production

(2009) J. Fraser Wright   HUMAN GENE THERAPY

Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

(2009) Yong Hong Chen et al.   NATURE MEDICINE

A chemical-genetic approach to study G protein regulation of β cell function in vivo

(2009) Jean-Marc Guettier et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA