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Journal Articles

Long-Term Real-World Safety and Efficacy of Liver-Directed AAV Gene Therapy for Severe Hemophilia with and without Inbibitors in Privately Owned Dogs

Bhavya S Doshi, Ben J Samelson-Jones, Timothy C Nichols, Elizabeth P Merricks, Valder R Arruda, Mary Beth Callan

Blood (2022) 140 (Supplement 1): 7780–7781.

https://doi.org/10.1182/blood-2022-164470

Published: 2022

Journal Articles

Multicenter, Randomized Crossover Trial Comparing Recombinant Von Willebrand Factor and Tranexamic Acid for Heavy Menstrual Bleeding in Von Willebrand Disease

Blood (2022) 140 (Supplement 1): 1413–1415.

https://doi.org/10.1182/blood-2022-156921

Published: 2022

Journal Articles

Ontogeny of the Alloimmune Anti-Canine Factor VIII Inhibitor Response in Severe Hemophilia Α Dogs

Robert Chen, Bhavya S Doshi, Timothy C. Nichols, Michael C. Milone, Ben J. Samelson-Jones, Valder R Arruda, Vijay Bhoj, Don L. Siegel

Blood (2021) 138 (Supplement 1): 3173.

https://doi.org/10.1182/blood-2021-149215

Published: 2021

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

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

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

Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII

Oscar A. Marcos-Contreras, Shannon M. Smith, Dwight A. Bellinger, Robin A. Raymer, Elizabeth Merricks, Armida Faella, Giulia Pavani, Shangzhen Zhou, Timothy C. Nichols, Katherine A. High, Paris Margaritis

Blood (2016) 127 (5): 565–571.

https://doi.org/10.1182/blood-2015-09-671420

Published: 2016

Includes: Supplemental data

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

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

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

Bad cholesterol breaking really bad

Timothy C. Nichols

Blood (2013) 122 (22): 3551–3553.

https://doi.org/10.1182/blood-2013-09-527697

Published: 2013

Journal Articles

The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy

Jonathan D. Finn, Timothy C. Nichols, Nikolaos Svoronos, Elizabeth P. Merricks, Dwight A. Bellenger, Shangshen Zhou, Paolo Simioni, Katherine A. High, Valder R. Arruda

Blood (2012) 120 (23): 4521–4523.

https://doi.org/10.1182/blood-2012-06-440123

Published: 2012

Journal Articles

Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs

Jennifer A. Dumont, Tongyao Liu, Susan C. Low, Xin Zhang, George Kamphaus, Paul Sakorafas, Cara Fraley, Douglas Drager, Thomas Reidy, Justin McCue, Helen W. G. Franck, Elizabeth P. Merricks, Timothy C. Nichols, Alan J. Bitonti, Glenn F. Pierce, Haiyan Jiang

Blood (2012) 119 (13): 3024–3030.

https://doi.org/10.1182/blood-2011-08-367813

Published: 2012

Includes: Supplemental data

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

Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide

Blood (2011) 118 (8): 2333–2341.

https://doi.org/10.1182/blood-2011-02-336172

Published: 2011

Includes: Supplemental data

NARROW

Long-Term Real-World Safety and Efficacy of Liver-Directed AAV Gene Therapy for Severe Hemophilia with and without Inbibitors in Privately Owned Dogs

Multicenter, Randomized Crossover Trial Comparing Recombinant Von Willebrand Factor and Tranexamic Acid for Heavy Menstrual Bleeding in Von Willebrand Disease

Ontogeny of the Alloimmune Anti-Canine Factor VIII Inhibitor Response in Severe Hemophilia Α Dogs

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

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

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

Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII

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

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

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

Bad cholesterol breaking really bad

The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy

Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs

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,

Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide

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American Society of Hematology

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American Society of Hematology

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