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Journal Articles

Specific Correction of the Intron-22 Inverted Factor VIII Gene in Autologous Blood Outgrowth Endothelial Cells from Patients with Severe Hemophilia A

Long V. Dinh, PhD, Eli Fine, PhD, Afshin Ameri, MD, Bernadette W. Luu, B.S., M.T. (CLS), Satish Kumar, PhD, Vincent P. Diego, PhD, Joanne E. Curran, PhD, Juan M Peralta, M.S., Henry Mead, M.B.A., M.P.H., Miguel A. Escobar, MD, Sarah Williams-Blangero, PhD, John Blangero, PhD, Jerry S. Powell, MD, Timothy C. Nichols, MD, Robert P. Hebbel, MD, Tom E. Howard, MD, PhD

Blood (2020) 136 (Supplement 1): 30–31.

https://doi.org/10.1182/blood-2020-142679

Published: 2020

Journal Articles

FVIII Protein Is Not Detectable in Human PBMCs or Livers from Dogs with an Intron-22 Inversion Mutation: Implications for FVIII Immunogenicity and Tolerance

Devi Gunasekera, MBBS, Pooja Vir, PhD, Ahmad Faisal Karim, PhD, Margaret V. Ragni, MD MPH, Elizabeth P. Merricks, PhD, Timothy C. Nichols, MD, Kathleen P. Pratt, PhD

Blood (2019) 134 (Supplement_1): 630.

https://doi.org/10.1182/blood-2019-124880

Published: 2019

Journal Articles

Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration

Giang N. Nguyen, BS, John K. Everett, PhD, Hayley Raymond, BA, Samita Kafle, BS, Elizabeth P. Merricks, PhD, Haig H. Kazazian, MD, Timothy C. Nichols, MD, Frederic D. Bushman, PhD, Denise E. Sabatino, PhD

Blood (2019) 134 (Supplement_1): 611.

https://doi.org/10.1182/blood-2019-126007

Published: 2019

Journal Articles

Hemophilia A Dogs Tolerant to Human Factor VIII Provide a Unique Model to Determine Efficacy and Safety of AAV Delivery of Novel Factor VIII Variants

Giang N. Nguyen, BS, Lauren E. Wimsey, DVM, Elizabeth P. Merricks, PhD, Katherine P. Ponder, MD, Timothy C. Nichols, MD, Denise E. Sabatino, PhD

Blood (2019) 134 (Supplement_1): 3628.

https://doi.org/10.1182/blood-2019-126085

Published: 2019

Journal Articles

Generation of a Unique Cohort of Hemophilia A Dogs Tolerant to Human FVIII for Evaluating the Safety and Efficacy of AAV Delivery of Wild Type and Variant Human FVIII

Giang N. Nguyen, Lauren Wimsey, Elizabeth Merricks, Katherine P. Ponder, MD, Timothy C. Nichols, MD, Denise E. Sabatino, PhD

Blood (2018) 132 (Supplement 1): 2453.

https://doi.org/10.1182/blood-2018-99-113492

Published: 2018

Journal Articles

The Role of Transgene Levels in Immune Tolerance Induction after AAV Gene Therapy in Hemophilia B Canine Models

Ben J. Samelson-Jones, MD PhD, Robert Andrew French, BS, Julie M. Crudele, PhD, Timothy C. Nichols, MD, Valder R Arruda, MD PhD

Blood (2017) 130 (Supplement 1): 2057.

https://doi.org/10.1182/blood.V130.Suppl_1.2057.2057

Published: 2017

Journal Articles

Liver-Directed Gene Therapy for Hemophilia B with Immune Stealth Lentiviral Vectors

Alessio Cantore, Michela Milani, Andrea Annoni, Tongyao Liu, Sara Bartolaccini, Mauro Biffi, Fabio Russo, Robert Peters, PhD, Angelo Lombardo, Timothy C. Nichols, MD, Eduard Ayuso, Luigi Naldini

Blood (2017) 130 (Supplement 1): 605.

https://doi.org/10.1182/blood.V130.Suppl_1.605.605

Published: 2017

Journal Articles

Complete Correction of Severe Canine Hemophilia B By Skeletal Muscle Directed AAV-Based FIX-Padua Gene Therapy in Inhibitor-Prone Dogs

Robert French, Nicholas Martin, Timothy C. Nichols, MD, Glenn P Niemeyer, Clinton D. Lothrop, PhD DVM, Valder R Arruda, MD PhD

Blood (2015) 126 (23): 3487.

https://doi.org/10.1182/blood.V126.23.3487.3487

Published: 2015

Journal Articles

VKORc1 Is Under-Expressed in Skeletal Muscle of Humans, Dogs and Mice: Potential Implications for Ectopic Coagulation Factor Expression in Pre-Clinical and Therapeutic Applications

Courtney T Connolly, Armida Faella, Timothy C. Nichols, MD, Katherine A. High, MD, Valder R. Arruda, MD, PhD, Paris Margaritis, DPhil

Blood (2014) 124 (21): 1477.

https://doi.org/10.1182/blood.V124.21.1477.1477

Published: 2014

Journal Articles

Unexpected Role of PACE/Furin Cleavage Site in FVIII Biology: Implications for Hemophilia a Therapy

Joshua I Siner, Julie M Crudele, Courtney T Connolly, Shangzhen Zhou, MD, Elizabeth P. Merricks, PhD, Timothy C. Nichols, MD, Rodney M. Camire, PhD, Valder R. Arruda, MD PhD

Blood (2014) 124 (21): 105.

https://doi.org/10.1182/blood.V124.21.105.105

Published: 2014

Journal Articles

A Novel FVIIa Variant with Increased Potency and Duration of Effect Compared to Wildtype FVIIa. A Study in a Dog Model of Hemophilia A

Debra Pittman, MS, Stacey Weston, PhD, Kathleen Shields, BSc, Chuenlei Parng, PhD, Steven Arkin, MD, Ed Madison, PhD, Timothy C. Nichols, MD, Joachim Fruebis, PhD

Blood (2011) 118 (21): 2252.

https://doi.org/10.1182/blood.V118.21.2252.2252

Published: 2011

Journal Articles

Phase II Biologic Effects Trial of Recombinant Interleukin-11 (rhIL-11, Neumega) in Moderate or Mild Hemophilia A or Von Willebrand Disease Unable to Use DDAVP,

Margaret V. Ragni, MD, MPH, Enrico M. Novelli, MD, Anila Murshed, BS, Elizabeth P. Merricks, PhD, Mark T. Kloos, BS, Timothy C. Nichols, MD

Blood (2011) 118 (21): 3308.

https://doi.org/10.1182/blood.V118.21.3308.3308

Published: 2011

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

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

FIX-R338L (FIX Padua) as a Successful Alternative for the Treatment of Canine Severe Hemophilia B.

Jonathan D Finn, PhD, Paolo Simioni, MD, PhD, Nicholas Iacobelli, B.Sc., Shangzhen Zhou, Timothy C. Nichols, MD, Katherine A. High, MD, Valder R. Arruda, M.D., Ph.D.

Blood (2009) 114 (22): 694.

https://doi.org/10.1182/blood.V114.22.694.694

Published: 2009

Journal Articles

Factor VIII-Fc Fusion Protein Shows Extended Half-Life and Hemostatic Activity in Hemophilia A Dogs.

Jennifer A Dumont, PhD, George D Kamphaus, PhD, Cara Fraley, MS, Tamera Ashworth, BS, Helen Franck, BS, Elizabeth P. Merricks, BS, Timothy C. Nichols, MD, Alan J Bitonti, PhD

Blood (2009) 114 (22): 545.

https://doi.org/10.1182/blood.V114.22.545.545

Published: 2009

Journal Articles

Recombinant Interleukin-11 (rhIL-11) in Women with Refractory Menorrhagia and Von Willebrand Disease.

Rachel C. Jankowitz, MD, Margaret V. Ragni, MD, MPH, Holly L. Chapman, RN, Elizabeth P. Merricks, BS, Mark T. Kloos, BS, Aaron M. Dillow, BS, Helen Franck, BS, Timothy C. Nichols, MD

Blood (2008) 112 (11): 1210.

https://doi.org/10.1182/blood.V112.11.1210.1210

Published: 2008

Journal Articles

Induction of Immune Tolerance to Canine FVIII in Hemophilia a Dogs with Inhibitors Using AAV-Mediated Expression of Canine FVIII

Jonathan D Finn, PhD, Denise E Sabatino, PhD, Margareth C Ozelo, MD, Shangzhen Zhou, MD, David Lillicrap, MD, Haig H Kazazian, MD, Timothy C. Nichols, MD, Valder R. Arruda, M.D., Ph.D.

Blood (2008) 112 (11): 243.

https://doi.org/10.1182/blood.V112.11.243.243

Published: 2008

Journal Articles

Successful Production of Canine FVIII: Biochemical and Functional Characterization in Hemophilia A Dogs.

Christian Furlan Freguia, PhD, Denise E. Sabatino, PhD, Andrey Santos, BS, Amy Lange, BS, Raffaella Toso, PhD, Timothy C. Nichols, MD, Rodney M. Camire, PhD, Valder R. Arruda, MD, PhD

Blood (2007) 110 (11): 495.

https://doi.org/10.1182/blood.V110.11.495.495

Published: 2007

Journal Articles

Gene Transfer of Canine FVIIa Results in Partial Correction of Canine Hemophilia: A Model for FVIII/FIX Gene-Based Bypassing Agents.

Paris Margaritis, PhD, Elise Roy, MS, Harre D. Downey, BS, Shangzen Zhou, PhD, Elizabeth Merricks, Aaron Dillow, Timothy C. Nichols, MD, Katherine A. High, MD

Blood (2007) 110 (11): 195.

https://doi.org/10.1182/blood.V110.11.195.195

Published: 2007

NARROW

Specific Correction of the Intron-22 Inverted Factor VIII Gene in Autologous Blood Outgrowth Endothelial Cells from Patients with Severe Hemophilia A

FVIII Protein Is Not Detectable in Human PBMCs or Livers from Dogs with an Intron-22 Inversion Mutation: Implications for FVIII Immunogenicity and Tolerance

Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration

Hemophilia A Dogs Tolerant to Human Factor VIII Provide a Unique Model to Determine Efficacy and Safety of AAV Delivery of Novel Factor VIII Variants

Generation of a Unique Cohort of Hemophilia A Dogs Tolerant to Human FVIII for Evaluating the Safety and Efficacy of AAV Delivery of Wild Type and Variant Human FVIII

The Role of Transgene Levels in Immune Tolerance Induction after AAV Gene Therapy in Hemophilia B Canine Models

Liver-Directed Gene Therapy for Hemophilia B with Immune Stealth Lentiviral Vectors

Complete Correction of Severe Canine Hemophilia B By Skeletal Muscle Directed AAV-Based FIX-Padua Gene Therapy in Inhibitor-Prone Dogs

VKORc1 Is Under-Expressed in Skeletal Muscle of Humans, Dogs and Mice: Potential Implications for Ectopic Coagulation Factor Expression in Pre-Clinical and Therapeutic Applications

Unexpected Role of PACE/Furin Cleavage Site in FVIII Biology: Implications for Hemophilia a Therapy

A Novel FVIIa Variant with Increased Potency and Duration of Effect Compared to Wildtype FVIIa. A Study in a Dog Model of Hemophilia A

Phase II Biologic Effects Trial of Recombinant Interleukin-11 (rhIL-11, Neumega) in Moderate or Mild Hemophilia A or Von Willebrand Disease Unable to Use DDAVP,

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

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

FIX-R338L (FIX Padua) as a Successful Alternative for the Treatment of Canine Severe Hemophilia B.

Factor VIII-Fc Fusion Protein Shows Extended Half-Life and Hemostatic Activity in Hemophilia A Dogs.

Recombinant Interleukin-11 (rhIL-11) in Women with Refractory Menorrhagia and Von Willebrand Disease.

Induction of Immune Tolerance to Canine FVIII in Hemophilia a Dogs with Inhibitors Using AAV-Mediated Expression of Canine FVIII

Successful Production of Canine FVIII: Biochemical and Functional Characterization in Hemophilia A Dogs.

Gene Transfer of Canine FVIIa Results in Partial Correction of Canine Hemophilia: A Model for FVIII/FIX Gene-Based Bypassing Agents.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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