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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

Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

Jonathan D. Finn, Margareth C. Ozelo, Denise E. Sabatino, Helen W. G. Franck, Elizabeth P. Merricks, Julie M. Crudele, Shangzhen Zhou, Haig H. Kazazian, David Lillicrap, Timothy C. Nichols, Valder R. Arruda

Blood (2010) 116 (26): 5842–5848.

https://doi.org/10.1182/blood-2010-06-288001

Published: 2010

Journal Articles

Successful Long Term Therapeutic Expression of Factor VIII in Hemophilia A Dogs After Administration of AAV-cFVIII Using a Two-Chain or Single Chain Delivery Approach.

Denise E. Sabatino, Ph.D., Ekaterina Altynova, B.S., Amy M. Lange, B.S., Shangzhen Zhou, M.D., Elizabeth P. Merricks, B.S., Aaron M. Dillow, B.S., Timothy C. Nichols, M.D., Valder R. Arruda, M.D., Ph.D., Haig H. Kazazian, Jr., M.D.

Blood (2009) 114 (22): 546.

https://doi.org/10.1182/blood.V114.22.546.546

Published: 2009

Journal Articles

Recombinant canine B-domain–deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model

Denise E. Sabatino, Christian Furlan Freguia, Raffaella Toso, Andrey Santos, Elizabeth P. Merricks, Haig H. Kazazian, Jr, Timothy C. Nichols, Rodney M. Camire, Valder R. Arruda

Blood (2009) 114 (20): 4562–4565.

https://doi.org/10.1182/blood-2009-05-220327

Published: 2009

Includes: Supplemental data

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

Long Term Dose-Dependent Correction of Hemophilia A Dogs Using AAV-8 and AAV-9-Mediated FVIII Gene Transfer.

Denise E. Sabatino, PhD, Amy M. Lange, Melinda Mucci, Rita Sarkar, PhD, Aaron M. Dillow, Timothy C. Nichols, MD, Valder R. Arruda, MD, Haig H. Kazazian, MD

Blood (2006) 108 (11): 999.

https://doi.org/10.1182/blood.V108.11.999.999

Published: 2006

Journal Articles

Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype

Rita Sarkar, Renee Tetreault, Guangping Gao, Lili Wang, Peter Bell, Randy Chandler, James M. Wilson, Haig H. Kazazian, Jr

Blood (2004) 103 (4): 1253–1260.

https://doi.org/10.1182/blood-2003-08-2954

Published: 2004

Journal Articles

Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII

Ciaran D. Scallan, Tongyao Liu, Amy E. Parker, Susannah L. Patarroyo-White, Haifeng Chen, Haiyan Jiang, Joseph Vargas, Dea Nagy, Sharon K. Powell, J. Fraser Wright, Rita Sarkar, Haig H. Kazazian, Alan McClelland, Linda B. Couto

Blood (2003) 102 (12): 3919–3926.

https://doi.org/10.1182/blood-2003-01-0222

Published: 2003

Journal Articles

Correction of the coagulation defect in hemophilia A mice through factor VIII expression in skin

Steven S. Fakharzadeh, Yue Zhang, Rita Sarkar, Haig H. Kazazian, Jr

Blood (2000) 95 (9): 2799–2805.

https://doi.org/10.1182/blood.V95.9.2799.009k23_2799_2805

Published: 2000

Journal Articles

The Molecular Basis for Cross-Reacting Material–Positive Hemophilia A Due to Missense Mutations Within the A2-Domain of Factor VIII

Kagehiro Amano, Rita Sarkar, Susan Pemberton, Geoffrey Kemball-Cook, Haig H. Kazazian, Jr, Randal J. Kaufman

Blood (1998) 91 (2): 538–548.

https://doi.org/10.1182/blood.V91.2.538

Published: 1998

NARROW

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

Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

Successful Long Term Therapeutic Expression of Factor VIII in Hemophilia A Dogs After Administration of AAV-cFVIII Using a Two-Chain or Single Chain Delivery Approach.

Recombinant canine B-domain–deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model

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

Long Term Dose-Dependent Correction of Hemophilia A Dogs Using AAV-8 and AAV-9-Mediated FVIII Gene Transfer.

Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype

Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII

Correction of the coagulation defect in hemophilia A mice through factor VIII expression in skin

The Molecular Basis for Cross-Reacting Material–Positive Hemophilia A Due to Missense Mutations Within the A2-Domain of Factor VIII

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American Society of Hematology

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American Society of Hematology

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