AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset

Enhancer viruses for combinatorial cell-subclass-specific labeling

(2021) Lucas T. Graybuck et al.   NEURON

Functional enhancer elements drive subclass-selective expression from mouse to primate neocortex

(2021) John K. Mich et al.   Cell Reports

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

(2020) Helena Costa Verdera 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

Human Immune Responses to Adeno-Associated Virus (AAV) Vectors

(2020) Giuseppe Ronzitti et al.   Frontiers in Immunology

Innovations present in the primate interneuron repertoire

(2020) Fenna M. Krienen et al.   NATURE

A community-based transcriptomics classification and nomenclature of neocortical cell types

(2020) Rafael Yuste et al.   NATURE NEUROSCIENCE

Viral manipulation of functionally distinct interneurons in mice, non-human primates and humans

(2020) Douglas Vormstein-Schneider et al.   NATURE NEUROSCIENCE

Systemic AAV vectors for widespread and targeted gene delivery in rodents

(2019) Rosemary C. Challis et al.   Nature Protocols

Adeno-associated virus 2 bound to its cellular receptor AAVR

(2019) Ran Zhang et al.   Nature Microbiology

TRIM9-Mediated Resolution of Neuroinflammation Confers Neuroprotection upon Ischemic Stroke in Mice

(2019) Jianxiong Zeng et al.   Cell Reports

From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1

(2019) Samiah A. Al-Zaidy et al.   PEDIATRIC NEUROLOGY

Immune Responses in the Liver

(2018) Paul Kubes et al.   Annual Review of Immunology

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

(2018) Moriel Zelikowsky et al.   CELL

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

Haemophilia gene therapy: From trailblazer to gamechanger

(2018) H. Evens et al.   HAEMOPHILIA

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

Severe Toxicity in Nonhuman Primates and Piglets with Systemic High-Dose Administration of Adeno-Associated Virus Serotype 9–Like Vectors: Putting Patients First

(2018) Terence R. Flotte et al.   HUMAN GENE THERAPY

Gene therapy clinical trials worldwide to 2017: An update

(2018) Samantha L. Ginn et al.   JOURNAL OF GENE MEDICINE

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

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Rapid and Complete Reversal of Sensory Ataxia by Gene Therapy in a Novel Model of Friedreich Ataxia

(2018) Françoise Piguet 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

A MicroRNA-Based Gene-Targeting Tool for Virally Labeling Interneurons in the Rodent Cortex

(2018) Marianna K. Keaveney et al.   Cell Reports

Assessment of Humoral, Innate, and T-Cell Immune Responses to Adeno-Associated Virus Vectors

(2018) Roberto Calcedo et al.   Human Gene Therapy Methods

Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain

(2017) Diane Golebiowski et al.   HUMAN GENE 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

Virus stamping for targeted single-cell infection in vitro and in vivo

(2017) Rajib Schubert et al.   NATURE BIOTECHNOLOGY

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

Basic liver immunology

(2016) Bin Gao   Cellular & Molecular Immunology

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

(2016) Michaël Hocquemiller et al.   HUMAN GENE THERAPY

AAVR: A Multi-Serotype Receptor for AAV

(2016) Candace Summerford 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

In vivo tissue-tropism of adeno-associated viral vectors

(2016) Arun Srivastava   Current Opinion in Virology

A Mammalian enhancer trap resource for discovering and manipulating neuronal cell types

(2016) Yasuyuki Shima et al.   eLife

Targeted CNS delivery using human MiniPromoters and demonstrated compatibility with adeno-associated viral vectors

(2014) Charles N de Leeuw et al.   Molecular Therapy-Methods & Clinical Development

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

(2013) F. Mingozzi et al.   BLOOD

Parkinson's Disease: Gene Therapies

(2013) P. G. Coune et al.   Cold Spring Harbor Perspectives in Medicine

Gene therapy for lipoprotein lipase deficiency

(2012) Daniel Gaudet et al.   CURRENT OPINION IN LIPIDOLOGY

Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates

(2012) Carmen Unzu et al.   Journal of Translational Medicine

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

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

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

Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

(2011) Lluis Samaranch et al.   HUMAN GENE THERAPY

TerminalN-Linked Galactose Is the Primary Receptor for Adeno-associated Virus 9

(2011) Shen Shen et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Peptide Ligands Incorporated into the Threefold Spike Capsid Domain to Re-Direct Gene Transduction of AAV8 and AAV9 In Vivo

(2011) Stefan Michelfelder et al.   PLoS One

Adeno-Associated Virus-Mediated Gene Transfer to Nonhuman Primate Liver Can Elicit Destructive Transgene-Specific T Cell Responses

(2009) Guangping Gao et al.   HUMAN GENE THERAPY

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

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY