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Journal Articles

Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

Nisha Nair, Dries De Wolf, Phuong Anh Nguyen, Quang Hong Pham, Ermira Samara-Kuko, Jeff Landau, Grant E. Blouse, Marinee K. Chuah, Thierry VandenDriessche

Blood (2021) 137 (21): 2902–2906.

https://doi.org/10.1182/blood.2020006005

Published: 2021

Includes: Supplemental data

Journal Articles

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

Markus Weiller, Helen Wang, Sogué Coulibaly, Maria Schuster, Hanspeter Rottensteiner, Kefeng Sun, Marinee K Chuah, Thierry Vandendriessche, Friedrich Scheiflinger, Werner Höllriegl

Blood (2019) 134 (Supplement_1): 4633.

https://doi.org/10.1182/blood-2019-124734

Published: 2019

Journal Articles

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

Nisha Nair, Melvin Y. Rincon, Hanneke Evens, Shilpita Sarcar, Sumitava Dastidar, Emira Samara-Kuko, Omid Ghandeharian, Hiu Man Viecelli, Beat Thöny, Pieter De Bleser, Thierry VandenDriessche, Marinee K. Chuah

Blood (2014) 123 (20): 3195–3199.

https://doi.org/10.1182/blood-2013-10-534032

Published: 2014

Includes: Supplemental data

Journal Articles

Targeting endothelial cells by gene therapy

Thierry VandenDriessche, Marinee K. Chuah

Blood (2013) 122 (12): 1993–1994.

https://doi.org/10.1182/blood-2013-08-518266

Published: 2013

Journal Articles

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

Alessio Cantore, Nisha Nair, Patrizia Della Valle, Mario Di Matteo, Janka Màtrai, Francesca Sanvito, Chiara Brombin, Clelia Di Serio, Armando D'Angelo, Marinee Chuah, Luigi Naldini, Thierry VandenDriessche

Blood (2012) 120 (23): 4517–4520.

https://doi.org/10.1182/blood-2012-05-432591

Published: 2012

Includes: Supplemental data

Journal Articles

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

Natalie J. Ward, Suzanne M. K. Buckley, Simon N. Waddington, Thierry VandenDriessche, Marinee K. L. Chuah, Amit C. Nathwani, Jenny McIntosh, Edward G. D. Tuddenham, Christine Kinnon, Adrian J. Thrasher, John H. McVey

Blood (2011) 117 (3): 798–807.

https://doi.org/10.1182/blood-2010-05-282707

Published: 2011

Includes: Supplemental data

Journal Articles

Muscling through AAV immunity

Thierry VandenDriessche

Blood (2009) 114 (10): 2009–2010.

https://doi.org/10.1182/blood-2009-07-228510

Published: 2009

Journal Articles

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

Thierry VandenDriessche, Zoltán Ivics, Zsuzsanna Izsvák, Marinee K. L. Chuah

Blood (2009) 114 (8): 1461–1468.

https://doi.org/10.1182/blood-2009-04-210427

Published: 2009

Journal Articles

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

Hideto Matsui, Masaru Shibata, Brian Brown, Andrea Labelle, Carol Hegadorn, Chandler Andrews, Marinee Chuah, Thierry VandenDriessche, Carol H. Miao, Christine Hough, David Lillicrap

Blood (2009) 114 (3): 677–685.

https://doi.org/10.1182/blood-2009-03-202267

Published: 2009

Journal Articles

FIXing bleeding joints

Thierry VandenDriessche

Blood (2008) 112 (12): 4366.

https://doi.org/10.1182/blood-2008-09-177220

Published: 2008

Journal Articles

Development of Megakaryocyte-Specific Lentiviral Vectors for Gene Therapy of Platelet Disorders.

Liesbeth De Waele, MD, Kathleen Freson, PhD, Chantal Thys, Christel Van Geet, MD, PhD, Désiré Collen, MD, PhD, Thierry VandenDriessche, PhD, Marinee Chuah, PhD

Blood (2007) 110 (11): 5143.

https://doi.org/10.1182/blood.V110.11.5143.5143

Published: 2007

Journal Articles

CAPitalizing on AAV

Thierry VandenDriessche

Blood (2006) 108 (1): 4–5.

https://doi.org/10.1182/blood-2006-04-016220

Published: 2006

Journal Articles

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

Simon F. De Meyer, Karen Vanhoorelbeke, Marinee K. Chuah, Inge Pareyn, Veerle Gillijns, Robert P. Hebbel, Désiré Collen, Hans Deckmyn, Thierry VandenDriessche

Blood (2006) 107 (12): 4728–4736.

https://doi.org/10.1182/blood-2005-09-3605

Published: 2006

Journal Articles

Phenotypic Correction of von Willebrand Disease Type 3 Blood-Derived Endothelial Cells with Lentiviral Vectors Expressing von Willebrand Factor.

Simon F. De Meyer, Karen Vanhoorelbeke, PhD, Marinee K. Chuah, PhD, Inge Pareyn, Veerle Gillijns, Robert P. Hebbel, MD, Hans Deckmyn, PhD, Thierry Vandendriessche, PhD

Blood (2005) 106 (11): 5522.

https://doi.org/10.1182/blood.V106.11.5522.5522

Published: 2005

Journal Articles

Liver gene transfer keeps immune system in check

Thierry VandenDriessche

Blood (2004) 104 (4): 910–911.

https://doi.org/10.1182/blood-2004-05-1977

Published: 2004

Journal Articles

Challenges and progress in gene therapy for hemophilia A

Thierry VandenDriessche

Blood (2003) 102 (6): 1938–1939.

https://doi.org/10.1182/blood-2003-07-2362

Published: 2003

Journal Articles

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

Marinee K. L. Chuah, Gudrun Schiedner, Lieven Thorrez, Brian Brown, Marion Johnston, Veerle Gillijns, Sabine Hertel, Nico Van Rooijen, David Lillicrap, Désiré Collen, Thierry VandenDriessche, Stefan Kochanek

Blood (2003) 101 (5): 1734–1743.

https://doi.org/10.1182/blood-2002-03-0823

Published: 2003

Journal Articles

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

Thierry VandenDriessche, Lieven Thorrez, Luigi Naldini, Antonia Follenzi, Lieve Moons, Zwi Berneman, Desire Collen, Marinee K. L. Chuah

Blood (2002) 100 (3): 813–822.

https://doi.org/10.1182/blood.V100.3.813

Published: 2002

Journal Articles

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

Marc Jacquemin, Renaud Lavend'homme, Abdellah Benhida, Beatrijs Vanzieleghem, Roseline d'Oiron, Jean-Maurice Lavergne, Hans H. Brackmann, Rainer Schwaab, Thierry VandenDriessche, Marinee K. L. Chuah, Marc Hoylaerts, Jean Guy G. Gilles, Kathelijne Peerlinck, Jos Vermylen, Jean-Marie R. Saint-Remy

Blood (2000) 96 (3): 958–965.

https://doi.org/10.1182/blood.V96.3.958

Published: 2000

NARROW

Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

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

Targeting endothelial cells by gene therapy

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

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

Muscling through AAV immunity

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

FIXing bleeding joints

Development of Megakaryocyte-Specific Lentiviral Vectors for Gene Therapy of Platelet Disorders.

CAPitalizing on AAV

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

Phenotypic Correction of von Willebrand Disease Type 3 Blood-Derived Endothelial Cells with Lentiviral Vectors Expressing von Willebrand Factor.

Liver gene transfer keeps immune system in check

Challenges and progress in gene therapy for hemophilia A

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

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

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

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American Society of Hematology

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ASH Publications

American Society of Hematology

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