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Journal Articles

Correction of Hemoglobin Levels in a Heterozygous Humanized Mouse Model of Thalassemia after Fetal Gene Therapy

Panicos Shangaris, MDMSc, Stavros P Loukogeorgakis, MDBSc PhDMRCS(Eng.), Sindhu Subramaniam, PhD, Mike Blundell, PhD, Nahla Bakhamis, MSc, Shanrun Liu, MD PhD, Simon Eaton, PhD, Durrgah Ramachandra, MSc, Panayiotis Maghsoudlou, BSc, Luca Urbani, PhD, Simon Waddington, MSc, PhD, Joy Archer, VMD PhDFRCPath DECVCP MRCVS, Mike Antoniou, PhD, Adrian J Thrasher, MD PhD, Thomas Ryan, PhD, Paolo De Coppi, MDPhD, Anna David, MDPhDMRCOG

Blood (2014) 124 (21): 3495.

https://doi.org/10.1182/blood.V124.21.3495.3495

Published: 2014

Journal Articles

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

Blood (2013) 121 (17): 3335–3344.

https://doi.org/10.1182/blood-2012-10-462200

Published: 2013

Includes: Supplemental data

Journal Articles

Assessing the Potential of Perinatal Gene Transfer Using Congenital Factor VII Deficiency as a Model System

Christopher J Binny, Jenny H McIntosh, Edward Tuddenham, MD, Simon Waddington, Suzanne Buckley, Adrian Thrasher, John T. Gray, PhD, Amit Nathwani, MD, PhD, Alice F Tarantal, PhD, Andrew Davidoff

Blood (2010) 116 (21): 247.

https://doi.org/10.1182/blood.V116.21.247.247

Published: 2010

Journal Articles

Stable High Level Coagulation Factor VIII Expression In Vivo Following Gene Transfer Using a Novel Expression Cassette Encoding a More Potent FVIII Variant

Jenny McIntosh, Peter J Lenting, Edward Tuddenham, MD, Motunrayo Sotannde, Simon Waddington, John T. Gray, PhD, Andrew Davidoff, Amit Nathwani, MD, PhD

Blood (2010) 116 (21): 250.

https://doi.org/10.1182/blood.V116.21.250.250

Published: 2010

Journal Articles

Functional characterization of a 13-bp deletion (c.-1522_-1510del13) in the promoter of the von Willebrand factor gene in type 1 von Willebrand disease

Maha Othman, Yvette Chirinian, Christine Brown, Colleen Notley, Nicholas Hickson, Daniel Hampshire, Suzanne Buckley, Simon Waddington, Alan L. Parker, Andrew Baker, Paula James, David Lillicrap

Blood (2010) 116 (18): 3645–3652.

https://doi.org/10.1182/blood-2009-12-261131

Published: 2010

NARROW

Correction of Hemoglobin Levels in a Heterozygous Humanized Mouse Model of Thalassemia after Fetal Gene Therapy

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

Assessing the Potential of Perinatal Gene Transfer Using Congenital Factor VII Deficiency as a Model System

Stable High Level Coagulation Factor VIII Expression In Vivo Following Gene Transfer Using a Novel Expression Cassette Encoding a More Potent FVIII Variant

Functional characterization of a 13-bp deletion (c.-1522_-1510del13) in the promoter of the von Willebrand factor gene in type 1 von Willebrand disease

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ASH Publications

American Society of Hematology

Correction of Hemoglobin Levels in a Heterozygous Humanized Mouse Model of Thalassemia after Fetal Gene Therapy

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

Assessing the Potential of Perinatal Gene Transfer Using Congenital Factor VII Deficiency as a Model System

Stable High Level Coagulation Factor VIII Expression In Vivo Following Gene Transfer Using a Novel Expression Cassette Encoding a More Potent FVIII Variant

Functional characterization of a 13-bp deletion (c.-1522_-1510del13) in the promoter of the von Willebrand factor gene in type 1 von Willebrand disease

Email alerts

ASH Publications

American Society of Hematology

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