BBB-crossing adeno-associated virus vector: An excellent gene delivery tool for CNS disease treatment

Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans

(2020) Gwladys Gernoux et al.   MOLECULAR THERAPY

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

Recent advances in the analysis full/empty capsid ratio and genome integrity of adeno-associated virus (AAV) gene delivery vectors

(2020) L. Hajba et al.   CURRENT MOLECULAR MEDICINE

Gene therapy for hemophilia: anticipating the unexpected

(2020) Glenn F. Pierce   Blood Advances

Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration

(2020) Scott H. Greenwald et al.   Molecular Therapy-Methods & Clinical Development

AAV-Mediated ApoC2 Gene Therapy: Reversal of Severe Hypertriglyceridemia and Rescue of Neonatal Death in ApoC2-Deficient Hamsters

(2020) Chun Yang et al.   Molecular Therapy-Methods & Clinical Development

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

Adeno-associated Virus (AAV) versus Immune Response

(2019) Joseph Rabinowitz et al.   Viruses-Basel

Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality

(2019) Eloise Hudry et al.   NEURON

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

rAAV-based brain slice culture models of Alzheimer’s and Parkinson’s disease inclusion pathologies

(2019) Cara L. Croft et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Polarized AAVR expression determines infectivity by AAV gene therapy vectors

(2019) Bradley A. Hamilton et al.   GENE THERAPY

Astrocyte-selective AAV gene therapy through the endogenous GFAP promoter results in robust transduction in the rat spinal cord following injury

(2019) Jarred M. Griffin et al.   GENE THERAPY

Blood–Brain Barrier in Cerebral Malaria: Pathogenesis and Therapeutic Intervention

(2019) Gopala Nishanth et al.   TRENDS IN PARASITOLOGY

Structure of the gene therapy vector, adeno-associated virus with its cell receptor, AAVR

(2019) Nancy L Meyer et al.   eLife

Hypoxia-enhanced Blood-Brain Barrier Chip recapitulates human barrier function and shuttling of drugs and antibodies

(2019) Tae-Eun Park et al.   Nature Communications

Standard screening methods underreport AAV-mediated transduction and gene editing

(2019) Jonathan F. Lang et al.   Nature Communications

Targeted Transgene Activation in the Brain Tissue by Systemic Delivery of Engineered AAV1 Expressing CRISPRa

(2019) Cia-Hin Lau et al.   Molecular Therapy-Nucleic Acids

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

(2019) Allison M. Keeler et al.   Annual Review of Virology

Capsid Engineering Overcomes Barriers Toward Adeno-Associated Virus Vector-Mediated Transduction of Endothelial Cells

(2019) L. Zhang et al.   HUMAN GENE THERAPY

Molecular pathology of neurodegenerative diseases: principles and practice

(2019) Gabor G Kovacs   JOURNAL OF CLINICAL PATHOLOGY

AAVR-Displaying Interfaces: Serotype-Independent Adeno-Associated Virus Capture and Local Delivery Systems

(2019) Seung-Hyun Kim et al.   Molecular Therapy-Nucleic Acids

B cells in autoimmune and neurodegenerative central nervous system diseases

(2019) Joseph J. Sabatino et al.   NATURE REVIEWS NEUROSCIENCE

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

(2019) Qin Huang et al.   PLoS One

Divergent engagements between adeno-associated viruses with their cellular receptor AAVR

(2019) Ran Zhang et al.   Nature Communications

Nanomaterial-based blood-brain-barrier (BBB) crossing strategies

(2019) Jinbing Xie et al.   BIOMATERIALS

Ly6a Differential Expression in Blood–Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B

(2019) Ana Rita Batista et al.   HUMAN GENE THERAPY

Selection of an Efficient AAV Vector for Robust CNS Transgene Expression

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

Central vein sign differentiates Multiple Sclerosis from central nervous system inflammatory vasculopathies

(2018) Pietro Maggi et al.   ANNALS OF NEUROLOGY

Blood-brain barrier shuttle peptides enhance AAV transduction in the brain after systemic administration

(2018) Xintao Zhang et al.   BIOMATERIALS

The Blood Brain Barrier: A Review of its Anatomy and Physiology in Health and Disease

(2018) Yousra Sharif et al.   CLINICAL ANATOMY

Personalized nanomedicine for CNS diseases

(2018) Ajeet Kaushik et al.   DRUG DISCOVERY TODAY

Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe’s Disease after Global Gene Therapy

(2018) Michael S. Marshall et al.   MOLECULAR THERAPY

Taking a Hint from Structural Biology: To Better Understand AAV Transport across the BBB

(2018) Dan Wang et al.   MOLECULAR THERAPY

Mapping the Structural Determinants Required for AAVrh.10 Transport across the Blood-Brain Barrier

(2018) Blake H. Albright et al.   MOLECULAR THERAPY

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

Triple Vectors Expand AAV Transfer Capacity in the Retina

(2018) Andrea Maddalena et al.   MOLECULAR THERAPY

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

(2018) Dan Wang et al.   Molecular Therapy-Methods & Clinical Development

Branched BBB-shuttle peptides: chemoselective modification of proteins to enhance blood–brain barrier transport

(2018) Cristina Díaz-Perlas et al.   Chemical Science

Emerging Technologies for Delivery of Biotherapeutics and Gene Therapy Across the Blood–Brain Barrier

(2018) Danica B. Stanimirovic et al.   BIODRUGS

Neuroinflammation in the central nervous system: Symphony of glial cells

(2018) Qiao‐qiao Yang et al.   GLIA

Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer

(2018) Dan Wang et al.   Molecular Therapy-Methods & Clinical Development

Sodium taurocholate cotransporting polypeptide is the limiting host factor of hepatitis B virus infection in macaque and pig hepatocytes

(2017) Florian A. Lempp et al.   HEPATOLOGY

Adeno-associated Virus (AAV) Serotypes Have Distinctive Interactions with Domains of the Cellular AAV Receptor

(2017) Sirika Pillay et al.   JOURNAL OF VIROLOGY

Intracranial AAV-IFN-β gene therapy eliminates invasive xenograft glioblastoma and improves survival in orthotopic syngeneic murine model

(2017) Dwijit GuhaSarkar et al.   Molecular Oncology

Progress toward Gene Therapy for Duchenne Muscular Dystrophy

(2017) Joel R. Chamberlain 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

AAV-mediated direct in vivo CRISPR screen identifies functional suppressors in glioblastoma

(2017) Ryan D Chow et al.   NATURE NEUROSCIENCE

Review: The blood-brain barrier; protecting the developing fetal brain

(2017) Kate Goasdoué et al.   PLACENTA

Brain endothelial cell‐targeted gene therapy of neurovascular disorders

(2016) Serena Marchiò et al.   EMBO Molecular Medicine

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

Vectors and strategies for nonviral cancer gene therapy

(2016) Jessica Pahle et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Efficient Transduction of Corneal Stroma by Adeno-Associated Viral Serotype Vectors for Implications in Gene Therapy of Corneal Diseases

(2016) Yi Lu et al.   HUMAN GENE THERAPY

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

(2016) Michaël Hocquemiller et al.   HUMAN GENE THERAPY

Age-associated physiological and pathological changes at the blood–brain barrier: A review

(2016) Franciska Erdő et al.   JOURNAL OF CEREBRAL BLOOD FLOW AND METABOLISM

Adeno-associated virus (AAV) vectors in cancer gene therapy

(2016) Jorge L. Santiago-Ortiz et al.   JOURNAL OF CONTROLLED RELEASE

Trafficking of adeno-associated virus vectors across a model of the blood-brain barrier; a comparative study of transcytosis and transduction using primary human brain endothelial cells

(2016) Steven F. Merkel et al.   JOURNAL OF NEUROCHEMISTRY

Glucose Transporters at the Blood-Brain Barrier: Function, Regulation and Gateways for Drug Delivery

(2016) Simon G. Patching   MOLECULAR NEUROBIOLOGY

Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector

(2016) Sourav R Choudhury et al.   MOLECULAR THERAPY

AAVR: A Multi-Serotype Receptor for AAV

(2016) Candace Summerford et al.   MOLECULAR THERAPY

In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy

(2016) Sourav R Choudhury et al.   MOLECULAR THERAPY

Erratum: Corrigendum: An essential receptor for adeno-associated virus infection

(2016) S. Pillay et al.   NATURE

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

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

From blood–brain barrier to blood–brain interface: new opportunities for CNS drug delivery

(2016) William A. Banks   NATURE REVIEWS DRUG DISCOVERY

Recombinant Adeno Associated Viral (AAV) vector type 9 delivery of Ex1-Q138-mutant huntingtin in the rat striatum as a short-time model for in vivo studies in drug discovery

(2016) Ilaria Ceccarelli et al.   NEUROBIOLOGY OF DISEASE

A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons

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

Systemic delivery of adeno-associated viral vectors

(2016) Dongsheng Duan   Current Opinion in Virology

CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector

(2016) Giridhar Murlidharan et al.   Molecular Therapy-Nucleic Acids

Establishment and Dysfunction of the Blood-Brain Barrier

(2015) Zhen Zhao et al.   CELL

Gene therapy for neurological disorders

(2015) Chi-Jing Choong et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates

(2015) Citra N. Mattar et al.   FASEB JOURNAL

Systemic Vascular Transduction by Capsid Mutant Adeno-Associated Virus After Intravenous Injection

(2015) Daniel M. Lipinski et al.   HUMAN GENE THERAPY

Long-Term Correction of Sandhoff Disease Following Intravenous Delivery of rAAV9 to Mouse Neonates

(2015) Jagdeep S Walia et al.   MOLECULAR THERAPY

Gene therapy returns to centre stage

(2015) Luigi Naldini   NATURE

Strategies to improve delivery of anticancer drugs across the blood–brain barrier to treat glioblastoma

(2015) Rajneet K. Oberoi et al.   NEURO-ONCOLOGY

Blood–brain barrier in health and disease

(2015) Alon Friedman et al.   SEMINARS IN CELL & DEVELOPMENTAL BIOLOGY

The Molecular Constituents of the Blood–Brain Barrier

(2015) Brian Wai Chow et al.   TRENDS IN NEUROSCIENCES

Facilitation of Drug Transport across the Blood–Brain Barrier with Ultrasound and Microbubbles

(2015) Stephen Meairs   Pharmaceutics

Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10

(2014) Bin Yang et al.   MOLECULAR THERAPY

Gene Transfer Properties and Structural Modeling of Human Stem Cell-derived AAV

(2014) Laura J Smith et al.   MOLECULAR THERAPY

Efficient Production of Dual Recombinant Adeno-Associated Viral Vectors for Factor VIII Delivery

(2014) Qizhao Wang et al.   Human Gene Therapy Methods

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

(2013) S J Gray et al.   GENE THERAPY

A Single Intravenous rAAV Injection as Late as P20 Achieves Efficacious and Sustained CNS Gene Therapy in Canavan Mice

(2013) Seemin Seher Ahmed et al.   MOLECULAR THERAPY

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

(2013) Leszek Lisowski et al.   NATURE

Neurogenic neuroinflammation: inflammatory CNS reactions in response to neuronal activity

(2013) Dimitris N. Xanthos et al.   NATURE REVIEWS NEUROSCIENCE

Systemic Delivery of Tyrosine-Mutant AAV Vectors Results in Robust Transduction of Neurons in Adult Mice

(2013) Asako Iida et al.   Biomed Research International

The blood-brain barrier in health and disease

(2012) Richard Daneman   ANNALS OF NEUROLOGY

AAV9: Over the Fence and Into the Woods …

(2011) John R Forsayeth et al.   MOLECULAR THERAPY

Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System

(2011) Hongwei Zhang et al.   MOLECULAR THERAPY

Persistent expression of biologically active anti-HER2 antibody by AAVrh.10-mediated gene transfer

(2010) G Wang et al.   CANCER GENE THERAPY

Robust cardiomyocyte-specific gene expression following systemic injection of AAV: in vivo gene delivery follows a Poisson distribution

(2010) K-M R Prasad et al.   GENE THERAPY

Enhanced Long-Term Transduction and Multilineage Engraftment of Human Hematopoietic Stem Cells Transduced with Tyrosine-Modified Recombinant Adeno-Associated Virus Serotype 2

(2010) M. Ariel Kauss et al.   HUMAN GENE THERAPY

Negative impact of rAAV2 mediated expression of SOCS3 on the regeneration of adult retinal ganglion cell axons

(2010) M. Hellström et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

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

(2010) Jun Xie et al.   MOLECULAR THERAPY

Pericytes are required for blood–brain barrier integrity during embryogenesis

(2010) Richard Daneman et al.   NATURE

Controlling brain tumor growth by intraventricular administration of an AAV vector encoding IFN-β

(2009) D H Meijer et al.   CANCER GENE THERAPY

Comparison of Adeno-Associated Virus Pseudotype 1, 2, and 8 Vectors Administered by Intramuscular Injection in the Treatment of Murine Phenylketonuria

(2009) Alexandre Rebuffat et al.   HUMAN GENE THERAPY

AAV9: a potential blood-brain barrier buster

(2009) Fredric P Manfredsson et al.   MOLECULAR THERAPY

Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons

(2009) Sandra Duque et al.   MOLECULAR THERAPY

Systematic Evaluation of AAV Vectors for Liver directed Gene Transfer in Murine Models

(2009) Lili Wang et al.   MOLECULAR THERAPY

Preventing Growth of Brain Tumors by Creating a Zone of Resistance

(2008) Casey A Maguire et al.   MOLECULAR THERAPY

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

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

High-efficiency Transduction of the Mouse Retina by Tyrosine-mutant AAV Serotype Vectors

(2008) Hilda Petrs-Silva et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY