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Journal Articles

Gene Therapy Expressing Platelet-Derived Factor VIII for Correction of Hemophilia Α with a History of Inhibitors

David A. Wilcox, Myriam Armant, Lynn Malec, Qizhen Shi, Bryon D. Johnson, Huiqing Xu, Lily M. Du, Lisa J. Duffy, Frederic D. Bushman, James H. Jerkins, Peiman Hematti, Ruta Brazauskas, Shawn M. Jobe, Parameswaran N. Hari, Mary Eapen

Blood (2023) 142 (Supplement 1): 2249.

https://doi.org/10.1182/blood-2023-172845

Published: 2023

Journal Articles

A high-throughput sequencing test for diagnosing inherited bleeding, thrombotic, and platelet disorders

Ilenia Simeoni, Jonathan C. Stephens, Fengyuan Hu, Sri V. V. Deevi, Karyn Megy, Tadbir K. Bariana, Claire Lentaigne, Sol Schulman, Suthesh Sivapalaratnam, Minka J. A. Vries, Sarah K. Westbury, Daniel Greene, Sofia Papadia, Marie-Christine Alessi, Antony P. Attwood, Matthias Ballmaier, Gareth Baynam, Emilse Bermejo, Marta Bertoli, Paul F. Bray, Loredana Bury, Marco Cattaneo, Peter Collins, Louise C. Daugherty, Rémi Favier, Deborah L. French, Bruce Furie, Michael Gattens, Manuela Germeshausen, Cedric Ghevaert, Anne C. Goodeve, Jose A. Guerrero, Daniel J. Hampshire, Daniel P. Hart, Johan W. M. Heemskerk, Yvonne M. C. Henskens, Marian Hill, Nancy Hogg, Jennifer D. Jolley, Walter H. Kahr, Anne M. Kelly, Ron Kerr, Myrto Kostadima, Shinji Kunishima, Michele P. Lambert, Ri Liesner, José A. López, Rutendo P. Mapeta, Mary Mathias, Carolyn M. Millar, Amit Nathwani, Marguerite Neerman-Arbez, Alan T. Nurden, Paquita Nurden, Maha Othman, Kathelijne Peerlinck, David J. Perry, Pawan Poudel, Pieter Reitsma, Matthew T. Rondina, Peter A. Smethurst, William Stevenson, Artur Szkotak, Salih Tuna, Christel van Geet, Deborah Whitehorn, David A. Wilcox, Bin Zhang, Shoshana Revel-Vilk, Paolo Gresele, Daniel B. Bellissimo, Christopher J. Penkett, Michael A. Laffan, Andrew D. Mumford, Augusto Rendon, Keith Gomez, Kathleen Freson, Willem H. Ouwehand, Ernest Turro

Blood (2016) 127 (23): 2791–2803.

https://doi.org/10.1182/blood-2015-12-688267

Published: 2016

Includes: Supplemental data

Journal Articles

Megakaryocyte- and megakaryocyte precursor–related gene therapies

David A. Wilcox

Blood (2016) 127 (10): 1260–1268.

https://doi.org/10.1182/blood-2015-07-607937

Published: 2016

Journal Articles

High-level transgene expression in induced pluripotent stem cell–derived megakaryocytes: correction of Glanzmann thrombasthenia

Spencer K. Sullivan, Jason A. Mills, Sevasti B. Koukouritaki, Karen K. Vo, Randolph B. Lyde, Prasuna Paluru, Guoha Zhao, Li Zhai, Lisa M. Sullivan, Yuhuan Wang, Siddharth Kishore, Eyad Z. Gharaibeh, Michele P. Lambert, David A. Wilcox, Deborah L. French, Mortimer Poncz, Paul Gadue

Blood (2014) 123 (5): 753–757.

https://doi.org/10.1182/blood-2013-10-530725

Published: 2014

Includes: Supplemental data

Journal Articles

In Vivo Selection Of Genetically Manipulated Hematopoietic Stem Cells For Platelet Gene Therapy Of Hemophilia A

Qizhen Shi, Jocelyn A. Schroeder, David A. Wilcox, Robert R. Montgomery, Yingyu Chen

Blood (2013) 122 (21): 2329.

https://doi.org/10.1182/blood.V122.21.2329.2329

Published: 2013

Journal Articles

Tissue-Specific Transgene Expression in Induced Pluripotent Stem (iPS) Cell-Derived Megakaryocytes: Correction of Glanzmann Thrombasthenia (GT)

Spencer K. Sullivan, MD, Jason A. Mills, PhD, Li Zhai, MD, Prasuna Paluru, MS, Karen Vo, Michele Lambert, MD, Sevasti B. Koukouritaki, PhD, Juan Fang, MD, David A. Wilcox, PhD, Deborah L. French, PhD, Mortimer Poncz, MD, Paul Gadue, PhD

Blood (2012) 120 (21): 387.

https://doi.org/10.1182/blood.V120.21.387.387

Published: 2012

Journal Articles

Disaggregation Signals Initiated by Ligand Binding and Platelet Aggregation

Cunji Gao, MD, PhD, Juan Fang, MD, Huiying Zhi, PhD, David A. Wilcox, PhD, Peter J. Newman, PhD

Blood (2011) 118 (21): 2196.

https://doi.org/10.1182/blood.V118.21.2196.2196

Published: 2011

Journal Articles

Heparin promotes platelet responsiveness by potentiating αIIbβ3-mediated outside-in signaling

Cunji Gao, Brian Boylan, Juan Fang, David A. Wilcox, Debra K. Newman, Peter J. Newman

Blood (2011) 117 (18): 4946–4952.

https://doi.org/10.1182/blood-2010-09-307751

Published: 2011

Includes: Supplemental data

Journal Articles

De Novo Synthesis & Storage of Human Factor VIII In Platelets Reduces Bleeding In Canine Hemophilia A

Lily M. Du, MD, Helen W.G. Franck, BS, Elizabeth P. Merricks, PhD, Paquita Nurden, MD, Eric S. Jensen, DVM, Sandra L. Haberichter, PhD, Troy B. Hawkins, PhD, Paula M. Jacobi, BS, Juan Fang, MD, Timothy C. Nichols, MD, Sevasti B. Koukouritaki, PhD, Qizhen Shi, MD, PhD, Robert R. Montgomery, MD, Kenneth Cornetta, MD, Alan T. Nurden, PhD, David A. Wilcox, PhD

Blood (2010) 116 (21): 2198.

https://doi.org/10.1182/blood.V116.21.2198.2198

Published: 2010

Journal Articles

The Important Role of Von Willebrand Factor In Platelet-Derived FVIII Gene Therapy of Murine Hemophilia A In the Presence of Inhibitors

Qizhen Shi, MD, PhD, Erin L. Kuether, Jocelyn A. Schroeder, Scot A. Fahs, David A. Wilcox, PhD, Robert R. Montgomery, MD

Blood (2010) 116 (21): 2201.

https://doi.org/10.1182/blood.V116.21.2201.2201

Published: 2010

Journal Articles

Platelet-Targeted Expression of Human BDD-FVIII Reduces Bleeding in Canine Hemophilia A.

Lily M. Du, MS, MD, Timothy C. Nichols, MD, Sandra L. Haberichter, PhD, Paula M. Jacobi, Eric S. Jensen, DVM, Juan Fang, MD, Qizhen Shi, MD, PhD, Robert R. Montgomery, MD, David A. Wilcox, PhD

Blood (2009) 114 (22): 691.

https://doi.org/10.1182/blood.V114.22.691.691

Published: 2009

Journal Articles

Sustained Phenotypic Correction of Murine Hemophilia A with Pre-Existing Anti-FVIII Immunity Using Lentivirus-Mediated Platelet-Specific FVIII Gene Transfer.

Qizhen Shi, MD, PhD, Erin L. Kuether, Brian C. Cooley, Ph.D., Scot A. Fahs, Jocelyn A. Schroeder, David A. Wilcox, PhD, Robert R. Montgomery, MD

Blood (2009) 114 (22): 29.

https://doi.org/10.1182/blood.V114.22.29.29

Published: 2009

Journal Articles

High Mortality in Mice with Platelets Expressing Integrin αIIbβ3 Lockedin Its High Affinity State.

David A. Wilcox, PhD, Juan Fang, MD, Paula North, MD, PhD, Paquita Nurden, MD, Alan T. Nurden, PhD, Nathalie Valentin, PhD

Blood (2008) 112 (11): 1832.

https://doi.org/10.1182/blood.V112.11.1832.1832

Published: 2008

Journal Articles

Recombinant Human HPA-1A Antibodies for Treatment of Fetomaternal Alloimmune Thrombocytopenia (FMAIT): Proof of Principle in An in Vivo Murine Model and Human Volunteer Studies

Cedric Ghevaert, David A. Wilcox, PhD, Juan Fang, Kathryn Armour, Mike R Clark, Willem H. Ouwehand, Lorna M Williamson

Blood (2008) 112 (11): 85.

https://doi.org/10.1182/blood.V112.11.85.85

Published: 2008

Journal Articles

Platelet-Targeted Expression of Coagulation Factor VIII (FVIII) Shows Efficacy for Using the Dog as a Large Animal Model for Gene Therapy of Hemophilia a

David A. Wilcox, PhD, Lily M. Du, MS, MD, Sandra L. Haberichter, PhD, Paula M. Jacobi, Juan Fang, MD, Eric S. Jensen, DVM, Qizhen Shi, MD, PhD, Robert R. Montgomery, MD

Blood (2008) 112 (11): 3525.

https://doi.org/10.1182/blood.V112.11.3525.3525

Published: 2008

Journal Articles

Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity

Qizhen Shi, Scot A. Fahs, David A. Wilcox, Erin L. Kuether, Patricia A. Morateck, Nicole Mareno, Hartmut Weiler, Robert R. Montgomery

Blood (2008) 112 (7): 2713–2721.

https://doi.org/10.1182/blood-2008-02-138214

Published: 2008

Journal Articles

Blockade of Chemotherapy-Induced Thrombocytopenia by Bax Inhibiting Peptides (BIPs) in Mouse Model.

Jose A. Gomez, MSc, Vivian Gama, MSc, Tomoyuki Yoshida, PhD, Michelle Stapleton, PhD, Cathy Paddock, MSc, David A. Wilcox, PhD, Peter J. Newman, PhD, Eimei Sato, PhD, Shigemi Matsuyama, PhD

Blood (2007) 110 (11): 281.

https://doi.org/10.1182/blood.V110.11.281.281

Published: 2007

Journal Articles

In the Presence of Pre-Existing Factor VIII (FVIII) Immunity, Hematopoietic Stem Cells (HSC) That Are Genetically Modified To Express FVIII in Platelets Were Successfully Transplanted into Hemophilic Mice under Myeloablative and Various Non-Myeloablative Conditions.

Qizhen Shi, Scot A. Fahs, David A. Wilcox, Erin L. Kuether, Hartmut Weiler, Robert R. Montgomery

Blood (2007) 110 (11): 768.

https://doi.org/10.1182/blood.V110.11.768.768

Published: 2007

Journal Articles

Endothelial and Platelet FVIII/VWF Expression - Divergence in Clinical Effect in Murine Models of Hemophilia A with and without FVIII Inhibitory Antibodies.

Qizhen Shi, Scot A. Fahs, David A. Wilcox, Hartmut Weiler, Sandra L. Haberichter, Robert R. Montgomery

Blood (2006) 108 (11): 3286.

https://doi.org/10.1182/blood.V108.11.3286.3286

Published: 2006

Journal Articles

Intravenous Immunoglobulin (IVIG) Diminishes Immune-Mediated Clearance of Platelets Expressing an Integrin αIIbβ3 Transgene Product That Restores Hemostasis in a Canine Model for Glanzmann Thrombasthenia.

Juan Fang, MD, Eric S. Jensen, DVM, Lily M. Du, MD,Ms, Mary K. Boudreaux, DVM,PhD, David A. Wilcox, PhD

Blood (2006) 108 (11): 3263.

https://doi.org/10.1182/blood.V108.11.3263.3263

Published: 2006

NARROW

Gene Therapy Expressing Platelet-Derived Factor VIII for Correction of Hemophilia Α with a History of Inhibitors

A high-throughput sequencing test for diagnosing inherited bleeding, thrombotic, and platelet disorders

Megakaryocyte- and megakaryocyte precursor–related gene therapies

High-level transgene expression in induced pluripotent stem cell–derived megakaryocytes: correction of Glanzmann thrombasthenia

In Vivo Selection Of Genetically Manipulated Hematopoietic Stem Cells For Platelet Gene Therapy Of Hemophilia A

Tissue-Specific Transgene Expression in Induced Pluripotent Stem (iPS) Cell-Derived Megakaryocytes: Correction of Glanzmann Thrombasthenia (GT)

Disaggregation Signals Initiated by Ligand Binding and Platelet Aggregation

Heparin promotes platelet responsiveness by potentiating αIIbβ3-mediated outside-in signaling

De Novo Synthesis & Storage of Human Factor VIII In Platelets Reduces Bleeding In Canine Hemophilia A

The Important Role of Von Willebrand Factor In Platelet-Derived FVIII Gene Therapy of Murine Hemophilia A In the Presence of Inhibitors

Platelet-Targeted Expression of Human BDD-FVIII Reduces Bleeding in Canine Hemophilia A.

Sustained Phenotypic Correction of Murine Hemophilia A with Pre-Existing Anti-FVIII Immunity Using Lentivirus-Mediated Platelet-Specific FVIII Gene Transfer.

High Mortality in Mice with Platelets Expressing Integrin αIIbβ3 Lockedin Its High Affinity State.

Recombinant Human HPA-1A Antibodies for Treatment of Fetomaternal Alloimmune Thrombocytopenia (FMAIT): Proof of Principle in An in Vivo Murine Model and Human Volunteer Studies

Platelet-Targeted Expression of Coagulation Factor VIII (FVIII) Shows Efficacy for Using the Dog as a Large Animal Model for Gene Therapy of Hemophilia a

Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity

Blockade of Chemotherapy-Induced Thrombocytopenia by Bax Inhibiting Peptides (BIPs) in Mouse Model.

In the Presence of Pre-Existing Factor VIII (FVIII) Immunity, Hematopoietic Stem Cells (HSC) That Are Genetically Modified To Express FVIII in Platelets Were Successfully Transplanted into Hemophilic Mice under Myeloablative and Various Non-Myeloablative Conditions.

Endothelial and Platelet FVIII/VWF Expression - Divergence in Clinical Effect in Murine Models of Hemophilia A with and without FVIII Inhibitory Antibodies.

Intravenous Immunoglobulin (IVIG) Diminishes Immune-Mediated Clearance of Platelets Expressing an Integrin αIIbβ3 Transgene Product That Restores Hemostasis in a Canine Model for Glanzmann Thrombasthenia.

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American Society of Hematology

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