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

Molecular therapy, 2014-07, Vol.22 (7), p.1299-1309

2014

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Canavan Disease as a Model for Gene Therapy-Mediated Myelin Repair

Frontiers in cellular neuroscience, 2021-04, Vol.15, p.661928-661928

2021

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Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties

Nature communications, 2020-06, Vol.11 (1), p.3279-3279, Article 3279

2020

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Editorial: Myelin Repair: At the Crossing-Lines of Myelin Biology and Gene Therapy

Frontiers in cellular neuroscience, 2022-02, Vol.16, p.853742-853742

2022

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Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice

Nature biotechnology, 2018-10, Vol.36 (9), p.839-842

2018

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Adeno-associated virus as a delivery vector for gene therapy of human diseases

Signal transduction and targeted therapy, 2024-04, Vol.9 (1), p.78-78, Article 78

2024

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Transcriptome Profiling of Neovascularized Corneas Reveals miR-204 as a Multi-target Biotherapy Deliverable by rAAVs

Molecular therapy. Nucleic acids, 2018-03, Vol.10, p.349-360

2018

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

Molecular therapy. Methods & clinical development, 2018-06, Vol.9, p.234-246

2018

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Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation

Molecular therapy. Methods & clinical development, 2023-09, Vol.30, p.303-314

2023

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Gene Therapy for Rare Neurological Disorders

Clinical pharmacology and therapeutics, 2022-04, Vol.111 (4), p.743-757

2022

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GammaTile ® : Surgically targeted radiation therapy for glioblastomas

Future oncology (London, England), 2020-10, Vol.16 (30), p.2445-2455

2020

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Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders

Methods in molecular biology (Clifton, N.J.), 2016, Vol.1382, p.429

2016

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Editorial. Lost in translation: recognition of the “ceiling effect” as a potential barrier to the success of neuroprotective strategies in spinal cord injury

Journal of neurosurgery. Spine, 2023-03, Vol.38 (3), p.297-298

2023

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GammaTile® (GT) as a brachytherapy platform for rapidly proliferating glioblastomas: from case series to clinical trials

Journal of neuro-oncology, 2024-02, Vol.166 (3), p.441-450

2024

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Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous System

Methods in molecular biology (Clifton, N.J.), 2019, Vol.1950, p.143

2019

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Reuben Matalon, MD, PhD, FACMG (1935–2021)

Human gene therapy, 2022-03, Vol.33 (5-6), p.221-222

2022

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Renewal of oligodendrocyte lineage reverses dysmyelination and CNS neurodegeneration through corrected N-acetylaspartate metabolism

Progress in neurobiology, 2023-07, Vol.226, p.102460-102460, Article 102460

2023

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Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery

Scientific reports, 2017-01, Vol.7 (1), p.40336-40336, Article 40336

2017

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Redirecting N -acetylaspartate metabolism in the central nervous system normalizes myelination and rescues Canavan disease

JCI insight, 2017-02, Vol.2 (3), p.e90807-e90807

2017

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GammaTile® brachytherapy in the treatment of recurrent glioblastomas

Neuro-oncology advances, 2022-01, Vol.4 (1), p.vdab185-vdab185

2022

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Editorial. Lost in translation: recognition of the "ceiling effect" as a potential barrier to the success of neuroprotective strategies in spinal cord injury

Journal of neurosurgery. Spine, 2022-11, Vol.38 (3), p.297

2022

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366. Hitting Two Birds with One Stone: How Efficacious Pre-Clinical Gene Therapy Cures Canavan Disease and Sheds Light onto the Pathomechanism

Molecular therapy, 2016-05, Vol.24, p.S147-S147

2016

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58. Pushing the Limits for Canavan Gene Therapy into Adulthood: Is There an Age Limit for Gene Therapy in CNS Disorders?

Molecular therapy, 2016-05, Vol.24, p.S25-S26

2016

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351. Efficacious Non-Oligodendrocyte Gene Therapy Suggests a New Dogma About CNS Compartmentalization of NAA Metabolism and Supports a Metabolic Sink Theory

Molecular therapy, 2016-05, Vol.24, p.S140-S141

2016

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349. The Cure of Canavan Disease: Is It a Scientific Fiction or Clinical Reality?

Molecular therapy, 2016-05, Vol.24, p.S140-S140

2016

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

Canavan Disease as a Model for Gene Therapy-Mediated Myelin Repair

Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties

Editorial: Myelin Repair: At the Crossing-Lines of Myelin Biology and Gene Therapy

Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice

Adeno-associated virus as a delivery vector for gene therapy of human diseases

Transcriptome Profiling of Neovascularized Corneas Reveals miR-204 as a Multi-target Biotherapy Deliverable by rAAVs

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

Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation

Gene Therapy for Rare Neurological Disorders

GammaTile ® : Surgically targeted radiation therapy for glioblastomas

Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders

Editorial. Lost in translation: recognition of the “ceiling effect” as a potential barrier to the success of neuroprotective strategies in spinal cord injury

GammaTile® (GT) as a brachytherapy platform for rapidly proliferating glioblastomas: from case series to clinical trials

Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous System

Reuben Matalon, MD, PhD, FACMG (1935–2021)

Renewal of oligodendrocyte lineage reverses dysmyelination and CNS neurodegeneration through corrected N-acetylaspartate metabolism

Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery

Redirecting N -acetylaspartate metabolism in the central nervous system normalizes myelination and rescues Canavan disease

GammaTile® brachytherapy in the treatment of recurrent glioblastomas

Editorial. Lost in translation: recognition of the "ceiling effect" as a potential barrier to the success of neuroprotective strategies in spinal cord injury

366. Hitting Two Birds with One Stone: How Efficacious Pre-Clinical Gene Therapy Cures Canavan Disease and Sheds Light onto the Pathomechanism

58. Pushing the Limits for Canavan Gene Therapy into Adulthood: Is There an Age Limit for Gene Therapy in CNS Disorders?

351. Efficacious Non-Oligodendrocyte Gene Therapy Suggests a New Dogma About CNS Compartmentalization of NAA Metabolism and Supports a Metabolic Sink Theory

349. The Cure of Canavan Disease: Is It a Scientific Fiction or Clinical Reality?

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