Recent progress and considerations for AAV gene therapies targeting the central nervous system

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

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

Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy

(2018) Rachel M. Bailey et al.   Molecular Therapy-Methods & Clinical Development

Intrathecal antibody distribution in the rat brain: surface diffusion, perivascular transport and osmotic enhancement of delivery

(2017) Michelle E. Pizzo et al.   JOURNAL OF PHYSIOLOGY-LONDON

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

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

Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion

(2017) Longping Victor Tse et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery

(2017) Sarah E. Sinnett et al.   Molecular Therapy-Methods & Clinical Development

Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome

(2017) Kamal K.E. Gadalla et al.   Molecular Therapy-Methods & Clinical Development

Adeno Associated Viral Vector Delivered RNAi for Gene Therapy of SOD1 Amyotrophic Lateral Sclerosis

(2016) Lorelei Stoica et al.   Frontiers in Molecular Neuroscience

Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery

(2016) Haiyan Fu et al.   Molecular Therapy-Methods & Clinical Development

Current developments in gene therapy for amyotrophic lateral sclerosis

(2015) Joseph M Scarrott et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

scAAVIL-1ra dosing trial in a large animal model and validation of long-term expression with repeat administration for osteoarthritis therapy

(2015) L R Goodrich et al.   GENE THERAPY

Emerging mechanisms of molecular pathology in ALS

(2015) Owen M. Peters et al.   JOURNAL OF CLINICAL INVESTIGATION

Multifactorial Gene Therapy Enhancing the Glutamate Uptake System and Reducing Oxidative Stress Delays Symptom Onset and Prolongs Survival in the SOD1-G93A ALS Mouse Model

(2015) Chen Benkler et al.   JOURNAL OF MOLECULAR NEUROSCIENCE

Structure of neurotropic adeno-associated virus AAVrh.8

(2015) Sujata Halder et al.   JOURNAL OF STRUCTURAL BIOLOGY

Comparative analyses of adeno-associated viral vector serotypes 1, 2, 5, 8 and 9 in marmoset, mouse and macaque cerebral cortex

(2015) Akiya Watakabe et al.   NEUROSCIENCE RESEARCH

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes

(2015) Daniel J Hui et al.   Molecular Therapy-Methods & Clinical Development

The structure of AAVrh32.33, a novel gene delivery vector

(2014) Kyle Mikals et al.   JOURNAL OF STRUCTURAL BIOLOGY

Adeno-Associated Virus Serotype 1 (AAV1)- and AAV5-Antibody Complex Structures Reveal Evolutionary Commonalities in Parvovirus Antigenic Reactivity

(2014) Yu-Shan Tseng et al.   JOURNAL OF VIROLOGY

Vector Design Tour de Force: Integrating Combinatorial and Rational Approaches to Derive Novel Adeno-associated Virus Variants

(2014) Damien Marsic et al.   MOLECULAR THERAPY

Adeno-Associated Virus Vectors and Neurological Gene Therapy

(2014) David S. Ojala et al.   NEUROSCIENTIST

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Comparative Efficacy and Safety of Multiple Routes of Direct CNS Administration of Adeno-Associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human Arylsulfatase A cDNA to Nonhuman Primates

(2014) Jonathan B. Rosenberg et al.   Human Gene Therapy Clinical Development

Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain

(2013) G P Swain et al.   GENE THERAPY

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

Effects of Immunosuppression on Circulating Adeno-Associated Virus Capsid-Specific T cells in Humans

(2013) Elizabeth M. Parzych et al.   HUMAN GENE THERAPY

Engraftment of a Galactose Receptor Footprint onto Adeno-associated Viral Capsids Improves Transduction Efficiency

(2013) Shen Shen et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

(2013) Virginia Haurigot et al.   JOURNAL OF CLINICAL INVESTIGATION

Structural Insights into Adeno-Associated Virus Serotype 5

(2013) L. Govindasamy et al.   JOURNAL OF VIROLOGY

Differential Adeno-Associated Virus Serotype-Specific Interaction Patterns with Synthetic Heparins and Other Glycans

(2013) M. Mietzsch et al.   JOURNAL OF VIROLOGY

Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS

(2013) Kevin D Foust et al.   MOLECULAR THERAPY

Analysis of Transduction Efficiency, Tropism and Axonal Transport of AAV Serotypes 1, 2, 5, 6, 8 and 9 in the Mouse Brain

(2013) Dominik F. Aschauer et al.   PLoS One

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

(2013) F. Mingozzi et al.   Science Translational Medicine

Directed evolution of novel adeno-associated viruses for therapeutic gene delivery

(2012) M A Bartel et al.   GENE THERAPY

Correction of Pathological Accumulation of Glycosaminoglycans in Central Nervous System and Peripheral Tissues of MPSIIIA Mice Through Systemic AAV9 Gene Transfer

(2012) Albert Ruzo et al.   HUMAN GENE THERAPY

Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease

(2012) Gabriele Mittermeyer et al.   HUMAN GENE THERAPY

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

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

Mapping a Neutralizing Epitope onto the Capsid of Adeno-Associated Virus Serotype 8

(2012) B. L. Gurda et al.   JOURNAL OF VIROLOGY

Long-Term Follow-Up After Gene Therapy for Canavan Disease

(2012) P. Leone et al.   Science Translational Medicine

Structure of adeno-associated virus-2 in complex with neutralizing monoclonal antibody A20

(2012) Dustin M. McCraw et al.   VIROLOGY

Long-Term Expression and Safety of Administration of AAVrh.10hCLN2 to the Brain of Rats and Nonhuman Primates for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis

(2012) Dolan Sondhi et al.   Human Gene Therapy Methods

Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents

(2011) Roberto Calcedo et al.   Clinical and Vaccine Immunology

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

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

Capsid-specific T-cell Responses to Natural Infections With Adeno-associated Viruses in Humans Differ From Those of Nonhuman Primates

(2011) Hua Li et al.   MOLECULAR THERAPY

Viral vectors for gene delivery to the central nervous system

(2011) Thomas B. Lentz et al.   NEUROBIOLOGY OF DISEASE

Structure–function analysis of receptor-binding in adeno-associated virus serotype 6 (AAV-6)

(2011) Qing Xie et al.   VIROLOGY

Development of the Blood-CSF Barrier

(2010) A. Statz et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Human Hepatocyte Growth Factor Receptor Is a Cellular Coreceptor for Adeno-Associated Virus Serotype 3

(2010) Chen Ling et al.   HUMAN GENE THERAPY

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

Structural Characterization of the Dual Glycan Binding Adeno-Associated Virus Serotype 6

(2010) R. Ng et al.   JOURNAL OF VIROLOGY

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

(2010) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Epidermal growth factor receptor is a co-receptor for adeno-associated virus serotype 6

(2010) Melodie L Weller et al.   NATURE MEDICINE

Memory T cells persisting within the brain after local infection show functional adaptations to their tissue of residence

(2010) L. M. Wakim et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The structure of adeno-associated virus serotype 3B (AAV-3B): Insights into receptor binding and immune evasion

(2010) Thomas F. Lerch et al.   VIROLOGY

Twinned crystals of adeno-associated virus serotype 3b prove suitable for structural studies

(2009) Thomas F. Lerch et al.   Acta Crystallographica Section F-Structural Biology and Crystallization Communications

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood–Brain Barrier (BBB)

(2009) Steven J Gray et al.   MOLECULAR THERAPY

Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle

(2009) Aravind Asokan et al.   NATURE BIOTECHNOLOGY

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

(2009) Yong Hong Chen et al.   NATURE MEDICINE

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

(2009) C. W. Christine et al.   NEUROLOGY

Adeno-associated virus-2 and its primary cellular receptor—Cryo-EM structure of a heparin complex

(2009) Jason O'Donnell et al.   VIROLOGY

Crystallization and preliminary X-ray structural studies of adeno-associated virus serotype 6

(2008) Qing Xie et al.   Acta Crystallographica Section F-Structural Biology and Crystallization Communications

Artificial Evolution with Adeno-Associated Viral Libraries

(2008) Hildegard Buning et al.   COMBINATORIAL CHEMISTRY & HIGH THROUGHPUT SCREENING

Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA

(2008) Stefan Worgall et al.   HUMAN GENE THERAPY

Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2–neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial

(2008) William J Marks et al.   LANCET NEUROLOGY

Expanded Repertoire of AAV Vector Serotypes Mediate Unique Patterns of Transduction in Mouse Brain

(2008) Cassia N Cearley et al.   MOLECULAR THERAPY

Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles

(2008) Wuping Li et al.   MOLECULAR THERAPY

DNA Shuffling of Adeno-associated Virus Yields Functionally Diverse Viral Progeny

(2008) James T Koerber et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Prolonged Susceptibility to Antibody-mediated Neutralization for Adeno-associated Vectors Targeted to the Liver

(2007) Samuel L Murphy et al.   MOLECULAR THERAPY