Management of Neuroinflammatory Responses to AAV-Mediated Gene Therapies for Neurodegenerative Diseases

AAV5-miHTT gene therapy demonstrates sustained huntingtin lowering and functional improvement in Huntington disease mouse models

(2019) Elisabeth A. Spronck et al.   Molecular Therapy-Methods & Clinical Development

Adeno-associated Virus (AAV) versus Immune Response

(2019) Joseph Rabinowitz et al.   Viruses-Basel

Prevalence of Anti-Adeno-Associated Virus Immune Responses in International Cohorts of Healthy Donors

(2019) Anita Kruzik et al.   Molecular Therapy-Methods & Clinical Development

Inflammation in CNS neurodegenerative diseases

(2018) Jodie Stephenson et al.   IMMUNOLOGY

Best practices for the use of intracerebroventricular drug delivery devices

(2018) Irene Slavc et al.   MOLECULAR GENETICS AND METABOLISM

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

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction

(2018) Zachary Fitzpatrick et al.   Molecular Therapy-Methods & Clinical Development

Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques

(2018) Juliette Hordeaux et al.   Molecular Therapy-Methods & Clinical Development

Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques

(2018) Juliette Hordeaux et al.   Molecular Therapy-Methods & Clinical Development

In vivo Imaging of Glial Activation in Alzheimer's Disease

(2018) Paul Edison et al.   Frontiers in Neurology

AAV gene delivery to the spinal cord: serotypes, methods, candidate diseases, and clinical trials

(2017) Nathan Hardcastle et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy

(2017) Hildegund C.J. Ertl et al.   HUMAN GENE THERAPY

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

(2017) Diane Golebiowski et al.   HUMAN GENE THERAPY

Clinical Gene Therapy for Neurodegenerative Diseases: Past, Present, and Future

(2017) Françoise Piguet et al.   HUMAN GENE THERAPY

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

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

Intracerebroventricular Delivery as a Safe, Long-Term Route of Drug Administration

(2017) Jessica L. Cohen-Pfeffer et al.   PEDIATRIC NEUROLOGY

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

(2017) Federico Mingozzi et al.   Annual Review of Virology

Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease

(2017) Manuela Corti et al.   Human Gene Therapy 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

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

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

How neuroinflammation contributes to neurodegeneration

(2016) R. M. Ransohoff   SCIENCE

Safety Evaluation of CNS Administered Biologics—Study Design, Data Interpretation, and Translation to the Clinic

(2016) Brian R. Vuillemenot et al.   TOXICOLOGICAL SCIENCES

Cerebellomedullary Cistern Delivery for AAV-Based Gene Therapy: A Technical Note for Nonhuman Primates

(2016) Lluis Samaranch et al.   Human Gene Therapy Methods

Targeted approaches to induce immune tolerance for Pompe disease therapy

(2016) Phillip A Doerfler et al.   Molecular Therapy-Methods & Clinical Development

Manufacturing of recombinant adeno-associated viral vectors for clinical trials

(2016) Nathalie Clément et al.   Molecular Therapy-Methods & Clinical Development

Microglia-specific targeting by novel capsid-modified AAV6 vectors

(2016) Awilda M Rosario et al.   Molecular Therapy-Methods & Clinical Development

Rapamycin increases neuronal survival, reduces inflammation and astrocyte proliferation after spinal cord injury

(2015) Yona Goldshmit et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

Widespread and Efficient Transduction of Spinal Cord and Brain Following Neonatal AAV Injection and Potential Disease Modifying Effect in ALS Mice

(2015) Jacob I Ayers et al.   MOLECULAR THERAPY

Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates

(2015) Kathrin Meyer et al.   MOLECULAR THERAPY

Neuroinflammation: Ways in Which the Immune System Affects the Brain

(2015) Richard M. Ransohoff et al.   Neurotherapeutics

Transduction efficiency of neurons and glial cells by AAV-1, -5, -9, -rh10 and -hu11 serotypes in rat spinal cord following contusion injury

(2014) H A Petrosyan et al.   GENE THERAPY

Biochemical, histological and functional correction of mucopolysaccharidosis Type IIIB by intra-cerebrospinal fluid gene therapy

(2014) Albert Ribera et al.   HUMAN MOLECULAR GENETICS

TLR2 and TLR9 Synergistically Control Herpes Simplex Virus Infection in the Brain

(2014) L. N. Sorensen et al.   JOURNAL OF IMMUNOLOGY

Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I

(2014) Christian Hinderer et al.   MOLECULAR THERAPY

Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia

(2014) Morgane Perdomini et al.   NATURE MEDICINE

Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse

(2014) Daniel J. Schuster et al.   Frontiers in Neuroanatomy

Biology of adeno-associated viral vectors in the central nervous system

(2014) Giridhar Murlidharan et al.   Frontiers in Molecular Neuroscience

Toll-Like Receptor Signaling Pathways

(2014) Takumi Kawasaki et al.   Frontiers in Immunology

Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating Mucopolysaccharidosis IIIB: Toxicology, Biodistribution, and Immunological Assessments in Primates

(2014) Darren A. Murrey et al.   Human Gene Therapy Clinical Development

Current perspectives on intrathecal drug delivery

(2014) Paul Christo et al.   Journal of Pain Research

B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study

(2014) M Corti et al.   Molecular Therapy-Methods & Clinical Development

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

3′UTRs take a long shot in the brain

(2013) Li Wang et al.   BIOESSAYS

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

(2013) F. Mingozzi et al.   BLOOD

Immune Responses to Intramuscular Administration of Alipogene Tiparvovec (AAV1-LPLS447X) in a Phase II Clinical Trial of Lipoprotein Lipase Deficiency Gene Therapy

(2013) Valerie Ferreira et al.   HUMAN GENE THERAPY

CpG-depleted adeno-associated virus vectors evade immune detection

(2013) Susan M. Faust et al.   JOURNAL OF CLINICAL INVESTIGATION

B-Cell Depletion and Immunomodulation before Initiation of Enzyme Replacement Therapy Blocks the Immune Response to Acid Alpha-Glucosidase in Infantile-Onset Pompe Disease

(2013) Melissa E. Elder et al.   JOURNAL OF PEDIATRICS

Plasmapheresis Eliminates the Negative Impact of AAV Antibodies on Microdystrophin Gene Expression Following Vascular Delivery

(2013) LG Chicoine et al.   MOLECULAR THERAPY

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

(2013) Paramita Chakrabarty et al.   PLoS One

Enlargement of Cerebral Ventricles as an Early Indicator of Encephalomyelitis

(2013) Stefano Lepore et al.   PLoS One

The Role of Markers of Inflammation in Traumatic Brain Injury

(2013) Thomas Woodcock et al.   Frontiers in Neurology

Decreased carbon-11-flumazenil binding in early Alzheimer’s disease

(2012) Belen Pascual et al.   BRAIN

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

(2012) Gabriele Mittermeyer et al.   HUMAN GENE THERAPY

Immunologic Privilege in the Central Nervous System and the Blood–Brain Barrier

(2012) Leslie L Muldoon et al.   JOURNAL OF CEREBRAL BLOOD FLOW AND METABOLISM

Innate immunity in the central nervous system

(2012) Richard M. Ransohoff et al.   JOURNAL OF CLINICAL INVESTIGATION

Cerebrospinal fluid and lumbar puncture: a practical review

(2012) Ben L. C. Wright et al.   JOURNAL OF NEUROLOGY

Proteasome Inhibition Is Partially Effective in Attenuating Pre-Existing Immunity against Recombinant Adeno-Associated Viral Vectors

(2012) Jozsef Karman et al.   PLoS One

TLR7 and TLR9 Trigger Distinct Neuroinflammatory Responses in the CNS

(2011) Niranjan B. Butchi et al.   AMERICAN JOURNAL OF PATHOLOGY

The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver

(2011) A. T. Martino et al.   BLOOD

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

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

A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8

(2011) Virginie Monteilhet et al.   MOLECULAR THERAPY

Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood–Brain Barrier Gene Delivery

(2011) Haiyan Fu et al.   MOLECULAR THERAPY

Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes

(2010) N Matthew Ellinwood et al.   MOLECULAR THERAPY

The myeloid cells of the central nervous system parenchyma

(2010) Richard M. Ransohoff et al.   NATURE

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

The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice

(2009) Jiangao Zhu et al.   JOURNAL OF CLINICAL INVESTIGATION

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

(2009) Sandra Duque et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

The Blood-Brain Barrier in Health and Chronic Neurodegenerative Disorders

(2008) Berislav V. Zlokovic   NEURON