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Gene and Cellular Therapies for Leukodystrophies

Pharmaceutics, 2023-10, Vol.15 (11), p.2522

2023

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Assessment of non-myelotoxic agents as a preparatory regimen for hematopoietic stem cell gene therapy

Human cell : official journal of Human Cell Research Society, 2024-10, Vol.38 (1), p.9, Article 9

2024

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Gene Therapy Developments for Pompe Disease

Biomedicines, 2022-01, Vol.10 (2), p.302

2022

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Recombination-activating gene 1 ( Rag1 )–deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome

Journal of allergy and clinical immunology, 2014-04, Vol.133 (4), p.1116-1123

2014

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Angiopoietin-like protein 3 promotes preservation of stemness during ex vivo expansion of murine hematopoietic stem cells

PloS one, 2014-08, Vol.9 (8), p.e105642-e105642

2014

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IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy

Molecular therapy. Methods & clinical development, 2022-12, Vol.27, p.109-130

2022

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Strategies to Genetically Modulate Dendritic Cells to Potentiate Anti-Tumor Responses in Hematologic Malignancies

Frontiers in immunology, 2018-05, Vol.9, p.982-982

2018

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Transplantation, gene therapy and intestinal pathology in MNGIE patients and mice

BMC gastroenterology, 2018-10, Vol.18 (1), p.149-149, Article 149

2018

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Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease

Molecular therapy. Methods & clinical development, 2020-06, Vol.17, p.1014-1025

2020

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Human post-mortem organotypic brain slice cultures: a tool to study pathomechanisms and test therapies

Acta neuropathologica communications, 2024-05, Vol.12 (1), p.83-17, Article 83

2024

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Gene Augmentation and Editing to Improve TCR Engineered T Cell Therapy against Solid Tumors

Vaccines (Basel), 2020-12, Vol.8 (4), p.733

2020

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Combining CRISPR-Cas9 and TCR exchange to generate a safe and efficient cord blood-derived T cell product for pediatric relapsed AML

Journal for immunotherapy of cancer, 2024-04, Vol.12 (4), p.e008174

2024

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Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease

Molecular therapy. Methods & clinical development, 2022-06, Vol.25, p.520-532

2022

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Efficient lentiviral transduction method to gene modify cord blood CD8+ T cells for cancer therapy applications

Molecular therapy. Methods & clinical development, 2021-06, Vol.21, p.357-368

2021

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Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype

Blood, 2010-07, Vol.115 (26), p.5329-5337

2010

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Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning

Molecular therapy, 2011-10, Vol.19 (10), p.1867-1877

2011

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Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE

Molecular therapy. Methods & clinical development, 2018-03, Vol.8 (C), p.152-165

2018

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In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice

Molecular therapy, 2024-05, Vol.32 (5), p.1328-1343

2024

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High-throughput analysis of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing

Molecular therapy, 2022-10, Vol.30 (10), p.3209-3225

2022

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Novel immortalized human fetal liver cell line, cBAL111, has the potential to differentiate into functional hepatocytes

BMC biotechnology, 2009-10, Vol.9 (1), p.89-89, Article 89

2009

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Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation

Journal of allergy and clinical immunology, 2018-09, Vol.142 (3), p.928-941.e8

2018

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Lentiviral gene therapy with IGF2-tagged GAA normalizes the skeletal muscle proteome in murine Pompe disease

Journal of proteomics, 2024-01, Vol.291, p.105037-105037, Article 105037

2024

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Correction of Griscelli Syndrome Type 2 causing mutations in the RAB27A gene with CRISPR/Cas9

Turkish journal of biology, 2024-01, Vol.48 (5), p.290-298

2024

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A “No‐Touch” Antibody‐Staining Method of Adherent Cells for High‐Throughput Flow Cytometry in 384‐Well Microplate Format for Cell‐Based Drug Library Screening

Cytometry. Part A, 2020-08, Vol.97 (8), p.845-851

2020

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Gene and Cellular Therapies for Leukodystrophies

Assessment of non-myelotoxic agents as a preparatory regimen for hematopoietic stem cell gene therapy

Gene Therapy Developments for Pompe Disease

Recombination-activating gene 1 ( Rag1 )–deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome

Angiopoietin-like protein 3 promotes preservation of stemness during ex vivo expansion of murine hematopoietic stem cells

IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy

Strategies to Genetically Modulate Dendritic Cells to Potentiate Anti-Tumor Responses in Hematologic Malignancies

Transplantation, gene therapy and intestinal pathology in MNGIE patients and mice

Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease

Human post-mortem organotypic brain slice cultures: a tool to study pathomechanisms and test therapies

Gene Augmentation and Editing to Improve TCR Engineered T Cell Therapy against Solid Tumors

Combining CRISPR-Cas9 and TCR exchange to generate a safe and efficient cord blood-derived T cell product for pediatric relapsed AML

Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease

Efficient lentiviral transduction method to gene modify cord blood CD8+ T cells for cancer therapy applications

Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype

Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning

Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE

In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice

High-throughput analysis of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing

Novel immortalized human fetal liver cell line, cBAL111, has the potential to differentiate into functional hepatocytes

Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation

Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations

Lentiviral gene therapy with IGF2-tagged GAA normalizes the skeletal muscle proteome in murine Pompe disease

Correction of Griscelli Syndrome Type 2 causing mutations in the RAB27A gene with CRISPR/Cas9

A “No‐Touch” Antibody‐Staining Method of Adherent Cells for High‐Throughput Flow Cytometry in 384‐Well Microplate Format for Cell‐Based Drug Library Screening

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