Characterization of AAV-mediated dorsal root ganglionopathy

The clinical landscape for AAV gene therapies

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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

(2021) John W Day et al.   LANCET NEUROLOGY

Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy

(2021) Jerry R. Mendell et al.   JAMA Neurology

Comparison of Different Liquid Chromatography-Based Purification Strategies for Adeno-Associated Virus Vectors

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SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS

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Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology

(2020) Juliette Hordeaux et al.   HUMAN GENE THERAPY

Safety of Direct Intraparenchymal AAVrh.10-mediated CNS Gene Therapy for Metachromatic Leukodystrophy

(2020) Jonathan B Rosenberg et al.   HUMAN GENE THERAPY

Current Clinical Applications of In Vivo Gene Therapy with AAVs

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AAVcis-regulatory sequences are correlated with ocular toxicity

(2019) Wenjun Xiong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Safe and sustained expression of human iduronidase after adeno-associated virus 9 intrathecal administration in infant rhesus monkeys

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Use of Nerve Conduction Assessments to Evaluate Drug-Induced Peripheral Neuropathy in Nonclinical Species—A Brief Review

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

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Sustained AAV9-mediated expression of a non-self protein in the CNS of non-human primates after immunomodulation

(2018) Arlene I. Ramsingh et al.   PLoS One

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

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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

A pilot study assessing T1-weighted muscle MRI in amyotrophic lateral sclerosis (ALS)

(2018) Luca Diamanti et al.   SKELETAL RADIOLOGY

Conventional and quantitative MRI in a novel feline model of demyelination and endogenous remyelination

(2018) Aaron S. Field et al.   JOURNAL OF MAGNETIC RESONANCE IMAGING

Toxicologic Pathology of the Peripheral Nervous System (PNS): Overview, Challenges, and Current Practices

(2018) Ingrid D. Pardo et al.   TOXICOLOGIC PATHOLOGY

Scalable Downstream Strategies for Purification of Recombinant Adeno- Associated Virus Vectors in Light of the Properties

(2015) Weihong Qu et al.   CURRENT PHARMACEUTICAL BIOTECHNOLOGY

Loss of intraepidermal nerve fiber density during SIV peripheral neuropathy is mediated by monocyte activation and elevated monocyte chemotactic proteins

(2015) Jessica R. Lakritz et al.   Journal of Neuroinflammation

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

AAV9-mediated Expression of a Non-self Protein in Nonhuman Primate Central Nervous System Triggers Widespread Neuroinflammation Driven by Antigen-presenting Cell Transduction

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Quantitative Evaluation and Visualization of Lumbar Foraminal Nerve Root Entrapment by Using Diffusion Tensor Imaging: Preliminary Results

(2011) Y. Eguchi et al.   AMERICAN JOURNAL OF NEURORADIOLOGY