M. Chuah

发表

Next-generation muscle-directed gene therapy by in silico vector design

G. Dickson, P. de Bleser, M. Sampaolesi, 2019, Nature Communications.

T. VandenDriessche, M. Chuah, 2013, Science Translational Medicine.

Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells

J. Joung, S. Seneca, J. Vermeesch, 2018, Nucleic acids research.

Angiogenesis enhances factor IX delivery and persistence from retrievable human bioengineered muscle implants.

H. Vandenburgh, J. Shansky, N. Callewaert, 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

Efficient stable gene transfer into human cells by the Sleeping Beauty transposon vectors.

Z. Izsvák, Z. Ivics, T. VandenDriessche, 2009, Methods.

Hitting the target without pulling the trigger.

T. VandenDriessche, M. Chuah, 2015, Molecular therapy : the journal of the American Society of Gene Therapy.

Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice.

T. VandenDriessche, M. Chuah, D. Collen, 1999, Proceedings of the National Academy of Sciences of the United States of America.

Preclinical gene therapy studies for hemophilia using adenoviral vectors.

Lieven Thorrez, T. VandenDriessche, M. Chuah, 2004, Seminars in thrombosis and hemostasis.

Liver-directed lentiviral gene therapy in a dog model of hemophilia B

L. Naldini, C. Doglioni, T. Nichols, 2015, Science Translational Medicine.

Efficiency of onco-retroviral and lentiviral gene transfer into primary mouse and human B-lymphocytes is pseudotype dependent.

W. Janssens, L. Naldini, T. VandenDriessche, 2003, Human gene therapy.

Gene therapy for hemophilia: hopes and hurdles

T. VandenDriessche, M. Chuah, D. Collen, 1998 .

induced pluripotent stem cells Emerging potential of transposons for gene therapy and generation of

Z. Izsvák, Z. Ivics, T. VandenDriessche, 2009 .

Treatment of phenylketonuria using minicircle‐based naked‐DNA gene transfer to murine liver

C. Harding, B. Thöny, S. Wong, 2014, Hepatology.

Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation

M. Y. Rincon, T. VandenDriessche, M. Chuah, 2015, Cardiovascular research.

Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nonmyeloablated immunodeficient mice.

T. VandenDriessche, M. Chuah, D. Collen, 2000, Human gene therapy.

Gene Therapy for Von Willebrand Disease

T. VandenDriessche, M. Chuah, I. Petrus, 2011 .

Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells

S. Benedetti, S. Dastidar, M. Loperfido, 2015, Nature Protocols.

Thrombospondin-2 prevents cardiac injury and dysfunction in viral myocarditis through the activation of regulatory T-cells.

P. Carmeliet, S. Heymans, J. D’hooge, 2012, Cardiovascular research.

Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors.

S. Kochanek, B. Brown, N. Van Rooijen, 2003, Blood.

Biosafety of adenoviral vectors.

T. VandenDriessche, M. Chuah, D. Collen, 2003, Current gene therapy.

Novel Hyperactive Transposons for Genetic Modification of Induced Pluripotent and Adult Stem Cells: A Nonviral Paradigm for Coaxed Differentiation

J. Vermeesch, Z. Izsvák, C. Verfaillie, 2010, Stem cells.

A murine model for induction of long-term immunologic tolerance to factor VIII does not require persistent detectable levels of plasma factor VIII and involves contributions from Foxp3+ T regulatory cells.

B. Brown, C. Miao, T. VandenDriessche, 2009, Blood.

Restoration of Plasma von Willebrand Factor Deficiency Is Sufficient to Correct Thrombus Formation After Gene Therapy for Severe von Willebrand Disease

P. Lenting, T. VandenDriessche, M. Chuah, 2008, Arteriosclerosis, thrombosis, and vascular biology.

In Vivo Induction of Type 1-Like Regulatory T Cells Using Genetically Modified B Cells Confers Long-Term IL-10-Dependent Antigen-Specific Unresponsiveness1

B. Weynand, W. Janssens, J. Vanoirbeek, 2009, The Journal of Immunology.

Antigen-Specific Unresponsiveness Confers Long-Term IL-10-Dependent T Cells Using Genetically Modified B Cells In Vivo Induction of Type 1-Like Regulatory and

B. Weynand, W. Janssens, J. Vanoirbeek, 2009 .

Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases

Rafael J. Yáñez-Muñoz, M. Zaslavskiy, F. Daboussi, 2012, Nucleic acids research.

Universal allogeneic CAR T cells engineered with Sleeping Beauty transposons and CRISPR-CAS9 for cancer immunotherapy

T. Gevaert, J. Tipanee, E. Samara-Kuko, 2022, Molecular therapy : the journal of the American Society of Gene Therapy.

Haemophilia gene therapy: From trailblazer to gamechanger

H. Evens, T. VandenDriessche, M. Chuah, 2018, Haemophilia : the official journal of the World Federation of Hemophilia.

Hemophilia Gene Therapy: Ready for Prime Time?

T. VandenDriessche, M. Chuah, 2017, Human gene therapy.

Absence of Thrombospondin-2 Causes Age-Related Dilated Cardiomyopathy

P. Carmeliet, W. Paulus, F. Van de Werf, 2009, Circulation.

Gene therapy strategies for hemophilia: benefits versus risks

T. VandenDriessche, M. Chuah, I. Petrus, 2010, The journal of gene medicine.

Viral vector-mediated gene therapy for hemophilia.

M. Chuah, D. Collen, T. Vandendriessche, 2001, Current gene therapy.

Phenotypic correction of von Willebrand disease type 3 blood-derived endothelial cells with lentiviral vectors expressing von Willebrand factor.

R. Hebbel, T. VandenDriessche, M. Chuah, 2006, Blood.

Recent Advances and Future Perspectives in Lentiviral Gene Therapy for Hemophilia A and B

T. VandenDriessche, M. Chuah, J. Mátrai, 2012 .

Inhibition of human immunodeficiency virus type-1 by retroviral vectors expressing antisense-TAR.

R. Morgan, B. Ensoli, M. Chuah, 1994, Human gene therapy.

Anti-viral strategies.

R. Morgan, M. Chuah, T. Vandendriessche, 1995, Molecular and cell biology of human diseases series.

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

D. Grimm, R. McKenna, M. Agbandje-McKenna, 2022, Science advances.

Getting Into the Rhythm With CRISPR.

T. VandenDriessche, M. Chuah, 2018, Circulation research.

therapy in hemophilic mice Hyperfunctional coagulation factor IX improves the efficacy of gene

C. Brombin, L. Naldini, F. Sanvito, 2013 .

in hemophilic mice Hyper-functional coagulation factor IX improves the efficacy of gene

Serio, L. Naldini, T. VandenDriessche, 2012 .

Bone marrow mesenchymal cells for haemophilia A gene therapy using retroviral vectors with modified long‐terminal repeats

F. Luyten, F. Dell’Accio, T. VandenDriessche, 2003, Haemophilia : the official journal of the World Federation of Hemophilia.

Glioblastoma: bridging the gap with gene therapy.

T. VandenDriessche, M. Chuah, 2013, The Lancet. Oncology.

Corrigendum: Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells

S. Benedetti, S. Dastidar, M. Loperfido, 2015, Nature Protocols.

Retroviral vectors induce epigenetic chromatin modifications and IL-10 production in transduced B cells via activation of toll-like receptor 2.

W. Janssens, T. VandenDriessche, M. Chuah, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

Challenges in Advancing the Field of Gene Therapy--American Society of Gene Therapy Stakeholders' Meeting. A critical review of the science, medicine and regulation.

M. Chuah, 2005, IDrugs : the investigational drugs journal.

Preclinical and clinical progress in hemophilia gene therapy

T. VandenDriessche, M. Chuah, J. Mátrai, 2010, Current opinion in hematology.

Moving forward toward a cure for hemophilia B.

T. VandenDriessche, M. Chuah, 2015, Molecular therapy : the journal of the American Society of Gene Therapy.

Genetic and Epigenetic Modification of Rat Liver Progenitor Cells via HNF4α Transduction and 5’ Azacytidine Treatment: An Integrated miRNA and mRNA Expression Profile Analysis

J. Bolleyn, T. Vanhaecke, V. Rogiers, 2020, Genes.

Vrije Universiteit Brussel Genetic and Epigenetic Modification of Rat Liver Progenitor Cells via HNF4α Transduction and 5’ Azacytidine Treatment: An Integrated miRNA and mRNA Expression Profile Analysis Bolleyn,

J. Bolleyn, T. Vanhaecke, V. Rogiers, 2020 .

PiggyBac toolbox.

T. VandenDriessche, M. Chuah, M. Di Matteo, 2012, Methods in molecular biology.

Codon optimization of human factor VIII cDNAs leads to high-level expression.

A. Thrasher, T. VandenDriessche, M. Chuah, 2011, Blood.

Clinical gene transfer studies for hemophilia A.

T. VandenDriessche, M. Chuah, D. Collen, 2004, Seminars in thrombosis and hemostasis.

Recent advances in lentiviral vector development and applications.

Janka Mátrai, Thierry VandenDriessche, T. VandenDriessche, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

Efficient Lentiviral Transduction and Improved Engraftment of Human Bone Marrow Mesenchymal Cells

H. Vandenburgh, F. Luyten, F. Dell’Accio, 2006, Stem cells.

Recent progress in gene therapy for hemophilia.

T. VandenDriessche, M. Chuah, N. Nair, 2012, Human gene therapy.

Clinical progress in gene therapy: sustained partial correction of the bleeding disorder in patients suffering from severe hemophilia B.

M. Chuah, T. Vandendriessche, 2012, Human gene therapy.

Hemophilia "A" gene therapy: Lost in translation.

T. VandenDriessche, M. Chuah, 2022, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene therapy for hemophilia

T. VandenDriessche, M. Chuah, D. Collen, 2001, The journal of gene medicine.

Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors

D. Grimm, W. Tulalamba, T. VandenDriessche, 2019, Gene Therapy.

Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy.

P. de Bleser, B. Thöny, S. Dastidar, 2014, Blood.

Platelet‐directed gene therapy overcomes inhibitory antibodies to factor VIII

M. Chuah, M. Chuah, T. Vandendriessche, 2012, Journal of thrombosis and haemostasis : JTH.

Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice.

P. Della Valle, C. Brombin, C. Di Serio, 2012, Blood.

Onco-retroviral and lentiviral vector-based gene therapy for hemophilia: preclinical studies.

T. VandenDriessche, M. Chuah, D. Collen, 2004, Seminars in thrombosis and hemostasis.

Bone marrow stromal cells as targets for gene therapy of hemophilia A.

H. Brems, T. VandenDriessche, M. Chuah, 1998, Human gene therapy.

Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice (gene therapyyretroviral vectorypseudotyping)

T. VandenDriessche, M. Chuah, D. Collen, 1999 .

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

Boris Jerchow, Janka Matrai, Zoltán Ivics, 2009, Nature Genetics.

A Calsequestrin Cis-Regulatory Motif Coupled to a Cardiac Troponin T Promoter Improves Cardiac Adeno-Associated Virus Serotype 9 Transduction Specificity.

A. Marian, T. Weber, R. Hajjar, 2018, Human gene therapy.

Repression of Cardiac Hypertrophy by KLF15: Underlying Mechanisms and Therapeutic Implications

J. Leenders, I. van der Made, Y. Pinto, 2012, PloS one.

Bone marrow stromal cells as targets for gene therapy.

M. Chuah, D. Collen, A. Van Damme, 2002, Current gene therapy.

Transposon-mediated gene transfer into adult and induced pluripotent stem cells.

S. Dastidar, T. VandenDriessche, M. Chuah, 2011, Current gene therapy.

Development and analysis of retroviral vectors expressing human factor VIII as a potential gene therapy for hemophilia A.

R. Morgan, T. VandenDriessche, M. Chuah, 1995, Human gene therapy.

Preclinical and clinical advances in transposon-based gene therapy

Y. C. Chai, J. Tipanee, T. VandenDriessche, 2017, Bioscience reports.

Recent developments in transposon-mediated gene therapy

T. VandenDriessche, M. Chuah, M. Di Matteo, 2012, Expert opinion on biological therapy.

Next-generation muscle-directed gene therapy by in silico vector design

G. Dickson, P. de Bleser, M. Sampaolesi, 2019, Nature Communications.

Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells.

Z. Izsvák, Z. Ivics, T. VandenDriessche, 2009, Blood.

Analysis of trans-dominant mutants of the HIV type 1 Rev protein for their ability to inhibit Rev function, HIV type 1 replication, and their use as anti-HIV gene therapeutics.

M. Daucher, R. Morgan, M. Chuah, 1995, AIDS research and human retroviruses.

Inhibition of clinical human immunodeficiency virus (HIV) type 1 isolates in primary CD4+ T lymphocytes by retroviral vectors expressing anti-HIV genes

R. Morgan, B. Ensoli, T. VandenDriessche, 1995, Journal of virology.

Atonal homolog 1 Is a Tumor Suppressor Gene

Bassem A. Hassan, P. Marynen, B. Hassan, 2009, PLoS biology.

Gene Therapy and Gene Editing for Myotonic Dystrophy

Y. C. Chai, S. Dastidar, T. VandenDriessche, 2019, Muscle Gene Therapy.

Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo.

L. Naldini, L. Moons, Z. Berneman, 2002, Blood.

Validation of miR-20a as a Tumor Suppressor Gene in Liver Carcinoma Using Hepatocyte-Specific Hyperactive piggyBac Transposons

J. V. Van Ginderachter, W. Tulalamba, J. Tipanee, 2020, Molecular therapy. Nucleic acids.

Transposons: Moving Forward from Preclinical Studies to Clinical Trials.

J. Tipanee, T. VandenDriessche, M. Chuah, 2017, Human gene therapy.

Comparative analysis of transposable element vector systems in human cells.

K. Kawakami, Z. Izsvák, Wei Chen, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

IL-10 Dampens TNF/Inducible Nitric Oxide Synthase-Producing Dendritic Cell-Mediated Pathogenicity during Parasitic Infection1

P. De Baetselier, M. Moser, M. Guilliams, 2009, The Journal of Immunology.

Genome-wide Computational Analysis Reveals Cardiomyocyte-specific Transcriptional Cis-regulatory Motifs That Enable Efficient Cardiac Gene Therapy

G. Dickson, P. de Bleser, T. Lahoutte, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

THROMBOSIS AND HEMOSTASIS Computationally designed liver-speci fi c transcriptional modules and hyperactive factor IX improve hepatic gene therapy

B. Thöny, S. Dastidar, S. Sarcar, 2015 .

Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

O. Christophe, P. de Bleser, M. Y. Rincon, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

COMPUTATIONALLY DESIGNED LIVER-SPECIFIC TRANSCRIPTIONAL CIS-REGULATORY MODULES AND HYPER-FUNCTIONAL FACTOR IX IMPROVE LIVER-TARGETED GENE THERAPY FOR HEMOPHILIA B

S. Sarcar, M. Y. Rincon, H. Evens, 2014 .

Hepatocyte-Targeted Expression by Integrase-Defective Lentiviral Vectors Induces Antigen-Specific Tolerance in Mice with Low Genotoxic Risk

C. von Kalle, A. Annoni, L. Naldini, 2011, Hepatology.

Xenogeneic liver models for gene therapy.

T. VandenDriessche, M. Chuah, 2010, Human gene therapy.

Efficacy and safety of adeno‐associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

J. Wilson, Lieven Thorrez, T. VandenDriessche, 2006, Journal of thrombosis and haemostasis : JTH.

Low hippocampal PI(4,5)P2 contributes to reduced cognition in old mice as a result of loss of MARCKS

R. D'Hooge, D. Balschun, C. Bagni, 2013, Nature Neuroscience.

Therapeutic levels of human Factor VIII in mice implanted with encapsulated cells: potential for gene therapy of haemophilia A

T. VandenDriessche, M. Chuah, G. Hortelano, 2002, The journal of gene medicine.

Optimizing delivery and expression of designer nucleases for genome engineering.

T. VandenDriessche, M. Chuah, 2013, Human gene therapy methods.

Moving gene therapy forward with mobile DNA.

T. VandenDriessche, M. Chuah, 2009, Human gene therapy.

Growth, differentiation, transplantation and survival of human skeletal myofibers on biodegradable scaffolds.

H. Vandenburgh, J. Shansky, D. Mooney, 2008, Biomaterials.

CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy.

T. VandenDriessche, M. Chuah, 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

Dose-dependent microdystrophin expression enhancement in cardiac muscle by a cardiac specific regulatory element.

L. Popplewell, G. Dickson, S. Jarmin, 2021, Human gene therapy.

Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases

J. Joung, C. Barbas, A. Bogdanove, 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9

S. Sarcar, M. Y. Rincon, H. Evens, 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

Differential effects of progenitor cell populations on left ventricular remodeling and myocardial neovascularization after myocardial infarction.

F. Van de Werf, F. Maes, J. Bogaert, 2010, Journal of the American College of Cardiology.

Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors

D. Grimm, W. Tulalamba, T. VandenDriessche, 2019, Gene Therapy.

Oncoretroviral and lentiviral vector-mediated gene therapy.

L. Naldini, T. VandenDriessche, M. Chuah, 2002, Methods in enzymology.

Hyperactive Factor IX Padua: A Game-Changer for Hemophilia Gene Therapy.

T. VandenDriessche, M. Chuah, 2018, Molecular therapy : the journal of the American Society of Gene Therapy.

Bone marrow mesenchymal cells for haemophilia A gene therapy using retroviral vectors with modified long‐terminal repeats

F. Dell’Accio, T. VandenDriessche, M. Chuah, 2003 .

Pluripotent Stem Cells for Gene Therapy of Hereditary Muscle Disorders

Y. C. Chai, D. Boon, T. VandenDriessche, 2019, Muscle Gene Therapy.

A novel cause of mild/moderate hemophilia A: mutations scattered in the factor VIII C1 domain reduce factor VIII binding to von Willebrand factor.

J. Vermylen, M. Hoylaerts, T. VandenDriessche, 2000, Blood.

Targeting endothelial cells by gene therapy.

T. VandenDriessche, M. Chuah, 2013, Blood.

493. Induction of Long-Term Immune Tolerance for Neo-Antigens Following Gene Therapy with High-Capacity Adenoviral Vectors by Transient Elimination of Kupffer Cells

S. Kochanek, N. Rooijen, G. Schiedner, 2003 .

Gene therapy for hemophilia

H. Evens, T. VandenDriessche, M. Chuah, 2001, The journal of gene medicine.

Preclinical and clinical gene therapy for haemophilia

T. VandenDriessche, M. Chuah, M. Chuah, 2004, Haemophilia : the official journal of the World Federation of Hemophilia.

Gene therapy for hemophilia

T. VandenDriessche, M. Chuah, D. Collen, 2001, Journal of Gene Medicine.

Solving the T-cell problem: generation of immune stealth AAV

K. High, T. VandenDriessche, M. Chuah, 2012 .

HEMOPHILIA GENE THERAPY: THE END OF THE BEGINNING?

T. VandenDriessche, M. Chuah, Kshitiz Singh, 2023, Human gene therapy.

Absence of Thrombospondin-2 Causes Age-Related Dilated Cardiomyopathy

P. Carmeliet, W. Paulus, B. Schroen, 2009, Circulation.

Retroviral vectors induce epigenetic chromatin modifications and IL-10 production in transduced B cells via activation of toll-like receptor 2.

W. Janssens, T. VandenDriessche, M. Chuah, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

C. Miao, T. VandenDriessche, M. Chuah, 2009, Blood.

Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice.

C. Brombin, C. Di Serio, L. Naldini, 2012, Blood.

Biosafety of onco-retroviral vectors.

T. VandenDriessche, M. Chuah, D. Collen, 2003, Current gene therapy.