Cell-Mediated Immunity to NAGLU Transgene Following Intracerebral Gene Therapy in Children With Mucopolysaccharidosis Type IIIB Syndrome

Molecular Bases of Neurodegeneration and Cognitive Decline, the Major Burden of Sanfilippo Disease

(2020) Rachel Heon-Roberts et al.   Journal of Clinical Medicine

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

(2020) Barbara A. Perez et al.   Brain Sciences

Battling Neurodegenerative Diseases with Adeno-Associated Virus-Based Approaches

(2020) Olja Mijanović et al.   Viruses-Basel

Sanfilippo Syndrome: Molecular Basis, Disease Models and Therapeutic Approaches

(2020) Noelia Benetó et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

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

(2019) Eloise Hudry et al.   NEURON

Engineering the AAV capsid to evade immune responses

(2019) Christopher Barnes et al.   CURRENT OPINION IN BIOTECHNOLOGY

Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle

(2019) Jérôme Poupiot 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

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

Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial

(2017) Marc Tardieu et al.   LANCET NEUROLOGY

The Balance between CD8 + T Cell-Mediated Clearance of AAV-Encoded Antigen in the Liver and Tolerance Is Dependent on the Vector Dose

(2017) Sandeep R.P. Kumar et al.   MOLECULAR THERAPY

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

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

Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency

(2017) Roberto Calcedo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

How neuroinflammation contributes to neurodegeneration

(2016) R. M. Ransohoff   SCIENCE

Pathogen-Specific T Cell Polyfunctionality Is a Correlate of T Cell Efficacy and Immune Protection

(2015) Anders Boyd et al.   PLoS One

Lymphocytes subsets reference values in childhood

(2014) F. Tosato et al.   CYTOMETRY PART A

Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human SGSH and SUMF1 cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial

(2014) Marc Tardieu et al.   HUMAN GENE THERAPY

Gene therapy for the neurological manifestations in lysosomal storage disorders

(2014) Seng H. Cheng   JOURNAL OF LIPID RESEARCH

Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions

(2014) Etiena Basner-Tschakarjan et al.   Frontiers in Immunology

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

Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression

(2013) Christian Mueller et al.   JOURNAL OF CLINICAL INVESTIGATION

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

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

Pediatric Reference Ranges for Proinflammatory and Anti-Inflammatory Cytokines in Cerebrospinal Fluid and Serum by Multiplexed Immunoassay

(2013) Michael R. Pranzatelli et al.   JOURNAL OF INTERFERON AND CYTOKINE RESEARCH

The Role of Markers of Inflammation in Traumatic Brain Injury

(2013) Thomas Woodcock et al.   Frontiers in Neurology

Innate immunity in the central nervous system

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

Multifunctional CD4 T Cell Responses in Patients with Active Tuberculosis

(2012) Zhengang Qiu et al.   Scientific Reports

Nonredundant Roles of IL-10 and TGF-β in Suppression of Immune Responses to Hepatic AAV-Factor IX Gene Transfer

(2011) Brad E Hoffman et al.   MOLECULAR THERAPY

Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and Greece

(2010) Bénédicte Héron et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Mucopolysaccharidosis type IIIB may predominantly present with an attenuated clinical phenotype

(2010) Marlies J. Valstar et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial

(2010) William J Marks et al.   LANCET NEUROLOGY

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

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

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

Polyfunctional HCV-specific T-cell responses are associated with effective control of HCV replication

(2008) Donatella Ciuffreda et al.   EUROPEAN JOURNAL OF IMMUNOLOGY

Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I

(2008) Patricia Dickson et al.   JOURNAL OF CLINICAL INVESTIGATION

Innate and adaptive immune activation in the brain of MPS IIIB mouse model

(2008) Julianne DiRosario et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Early Neurodegeneration Progresses Independently of Microglial Activation by Heparan Sulfate in the Brain of Mucopolysaccharidosis IIIB Mice

(2008) Jérôme Ausseil et al.   PLoS One