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Polycystin-1 Regulates Actomyosin Contraction and the Cellular Response to Extracellular Stiffness

Scientific reports, 2019-11, Vol.9 (1), p.16640-15, Article 16640

2019

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Isolation and characterization of tumorigenic, stem-like neural precursors from human glioblastoma

Cancer research (Chicago, Ill.), 2004-10, Vol.64 (19), p.7011-7021

2004

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Site-specific integration and tailoring of cassette design for sustainable gene transfer

Nature methods, 2011-10, Vol.8 (10), p.861-869

2011

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Novel bicistronic lentiviral vectors correct β-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: implications for in vivo and ex vivo gene therapy of GM2 gangliosidosis

Neurobiology of disease, 2020-02, Vol.134, p.104667-104667, Article 104667

2020

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Human neural stem cells ameliorate autoimmune encephalomyelitis in non-human primates

Annals of neurology, 2009-09, Vol.66 (3), p.343-354

2009

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In vivo Gene Therapy to the Liver and Nervous System: Promises and Challenges

Frontiers in medicine, 2022-01, Vol.8, p.774618-774618

2022

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Human iPSC-Based Models for the Development of Therapeutics Targeting Neurodegenerative Lysosomal Storage Diseases

Frontiers in molecular biosciences, 2020-09, Vol.7, p.224-224

2020

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Human iPSC-based models highlight defective glial and neuronal differentiation from neural progenitor cells in metachromatic leukodystrophy

Cell death & disease, 2018-06, Vol.9 (6), p.698-15, Article 698

2018

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Generation of Human Induced Pluripotent Stem Cell‐Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy

Stem cells translational medicine, 2017-02, Vol.6 (2), p.352-368

2017

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Integrated Computational Analysis Highlights unique miRNA Signatures in the Subventricular Zone and Striatum of GM2 Gangliosidosis Animal Models

International journal of molecular sciences, 2019-06, Vol.20 (13), p.3179

2019

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Delivery Platforms for CRISPR/Cas9 Genome Editing of Glial Cells in the Central Nervous System

Frontiers in genome editing, 2021-03, Vol.3, p.644319-644319

2021

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Two factor-based reprogramming of rodent and human fibroblasts into Schwann cells

Nature communications, 2017-02, Vol.8 (1), p.14088-14088, Article 14088

2017

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Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models

Human molecular genetics, 2010-06, Vol.19 (11), p.2208-2227

2010

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In vitro Validation of Chimeric β-Galactosylceramidase Enzymes With Improved Enzymatic Activity and Increased Secretion

Frontiers in molecular biosciences, 2020-07, Vol.7, p.167-167

2020

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Neural Stem Cell Gene Therapy Ameliorates Pathology and Function in a Mouse Model of Globoid Cell Leukodystrophy

Stem cells (Dayton, Ohio), 2011-10, Vol.29 (10), p.1559-1571

2011

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Dynamic Activity of miR-125b and miR-93 during Murine Neural Stem Cell Differentiation In Vitro and in the Subventricular Zone Neurogenic Niche

PloS one, 2013-06, Vol.8 (6), p.e67411-e67411

2013

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Robust in vivo Gene Transfer into Adult Mammalian Neural Stem Cells by Lentiviral Vectors

Proceedings of the National Academy of Sciences - PNAS, 2004-10, Vol.101 (41), p.14835-14840

2004

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Therapeutic advantages of combined gene/cell therapy strategies in a murine model of GM2 gangliosidosis

Molecular therapy. Methods & clinical development, 2022-06, Vol.25, p.170-189

2022

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The galactocerebrosidase enzyme contributes to maintain a functional neurogenic niche during early post-natal CNS development

Human molecular genetics, 2012-11, Vol.21 (21), p.4732-4750

2012

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Effects of developmental age, brain region, and time in culture on long-term proliferation and multipotency of neural stem cell populations

Journal of comparative neurology (1911), 2009-11, Vol.517 (3), p.333-349

2009

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Robust generation of oligodendrocyte progenitors from human neural stem cells and engraftment in experimental demyelination models in mice

PloS one, 2010-04, Vol.5 (4), p.e10145-e10145

2010

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Gene delivery targeted to oligodendrocytes using a lentiviral vector

The journal of gene medicine, 2014-11, Vol.16 (11-12), p.364-373

2014

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Epidermal and Fibroblast Growth Factors Behave as Mitogenic Regulators for a Single Multipotent Stem Cell-Like Population from the Subventricular Region of the Adult Mouse Forebrain

The Journal of neuroscience, 1999-05, Vol.19 (9), p.3287-3297

1999

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Perspective on innovative therapies for globoid cell leukodystrophy

Journal of neuroscience research, 2016-11, Vol.94 (11), p.1304-1317

2016

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Polycystin-1 Regulates Actomyosin Contraction and the Cellular Response to Extracellular Stiffness

Isolation and characterization of tumorigenic, stem-like neural precursors from human glioblastoma

Multicenter, randomised, open-label, non-comparative phase 2 trial on the efficacy and safety of the combination of bevacizumab and trabectedin with or without carboplatin in women with partially platinum-sensitive recurrent ovarian cancer

Site-specific integration and tailoring of cassette design for sustainable gene transfer

Novel bicistronic lentiviral vectors correct β-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: implications for in vivo and ex vivo gene therapy of GM2 gangliosidosis

Human neural stem cells ameliorate autoimmune encephalomyelitis in non-human primates

In vivo Gene Therapy to the Liver and Nervous System: Promises and Challenges

Human iPSC-Based Models for the Development of Therapeutics Targeting Neurodegenerative Lysosomal Storage Diseases

Human iPSC-based models highlight defective glial and neuronal differentiation from neural progenitor cells in metachromatic leukodystrophy

Generation of Human Induced Pluripotent Stem Cell‐Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy

Integrated Computational Analysis Highlights unique miRNA Signatures in the Subventricular Zone and Striatum of GM2 Gangliosidosis Animal Models

Delivery Platforms for CRISPR/Cas9 Genome Editing of Glial Cells in the Central Nervous System

Two factor-based reprogramming of rodent and human fibroblasts into Schwann cells

Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models

In vitro Validation of Chimeric β-Galactosylceramidase Enzymes With Improved Enzymatic Activity and Increased Secretion

Neural Stem Cell Gene Therapy Ameliorates Pathology and Function in a Mouse Model of Globoid Cell Leukodystrophy

Dynamic Activity of miR-125b and miR-93 during Murine Neural Stem Cell Differentiation In Vitro and in the Subventricular Zone Neurogenic Niche

Robust in vivo Gene Transfer into Adult Mammalian Neural Stem Cells by Lentiviral Vectors

Therapeutic advantages of combined gene/cell therapy strategies in a murine model of GM2 gangliosidosis

The galactocerebrosidase enzyme contributes to maintain a functional neurogenic niche during early post-natal CNS development

Effects of developmental age, brain region, and time in culture on long-term proliferation and multipotency of neural stem cell populations

Robust generation of oligodendrocyte progenitors from human neural stem cells and engraftment in experimental demyelination models in mice

Gene delivery targeted to oligodendrocytes using a lentiviral vector

Epidermal and Fibroblast Growth Factors Behave as Mitogenic Regulators for a Single Multipotent Stem Cell-Like Population from the Subventricular Region of the Adult Mouse Forebrain

Perspective on innovative therapies for globoid cell leukodystrophy

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