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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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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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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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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

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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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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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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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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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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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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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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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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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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Diseases caused by different mutations in the two alleles of a gene are treated in mice by Cas-9-induced allelic exchange: Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice

Nature biotechnology, 2018-08, Vol.36 (9), p.839-842

2018

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586. Development of Anti-Angiogenic miRNA Therapeutics for Corneal Neovascularization

Molecular therapy, 2015-05, Vol.23, p.S233-S233

2015

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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

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

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

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

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

Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders

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

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

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

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

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

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

Diseases caused by different mutations in the two alleles of a gene are treated in mice by Cas-9-induced allelic exchange: Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice

586. Development of Anti-Angiogenic miRNA Therapeutics for Corneal Neovascularization

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