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Journal Articles

Evaluating ROR1 As a Target in Childhood Leukemia

Paraskevi Diamanti, PhD, Bethan Kathleen Bailey, BSc, Obinna Ebere Iheanacho, MD, Amit C. Nathwani, MD PhD, Allison Blair, PhD

Blood (2019) 134 (Supplement_1): 2635.

https://doi.org/10.1182/blood-2019-126734

Published: 2019

Journal Articles

Liver-Directed AAV Gene Therapy for Gaucher Disease

Carlos J Miranda, PhD, Miriam Canavese, PhD, Elisa Chisari, MS, Jalpa Pandya, MPhil, Clement Cocita, PhD, Maria Portillo, MRes, Jenny McIntosh, PhD, Azadeh Kia, PhD, Jonathan H. Foley, PhD, Allison Dane, PhD, Jey M Jeyakumar, PhD, Rose Sheridan, PhD, Romuald Corbau, Amit C. Nathwani, MD PhD

Blood (2019) 134 (Supplement_1): 3354.

https://doi.org/10.1182/blood-2019-124280

Published: 2019

Journal Articles

Enhancing FVIII Expression By Modifying B-Domain Sequence for Hemophilia A Gene Therapy

Doyoung Lee, PhD, Jenny McIntosh, PhD, Allison Dane, PhD, Cecilia Rosales, PhD, Anne Riddell, Edward Tuddenham, MD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 3484.

https://doi.org/10.1182/blood-2018-99-113830

Published: 2018

Journal Articles

A Novel Lysine to Arginine Substitution at Position 301 Enhances Activity of Factor IX

Daniel Verhoef, Jonathan H. Foley, Andrew Goodale, Emma Macrae, Jenny McIntosh, PhD, Romuald Corbau, PhD, Keith Gomez, Anne Riddell, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 3772.

https://doi.org/10.1182/blood-2018-99-112832

Published: 2018

Journal Articles

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

Amit C Nathwani, MD PhD, Ulrike Reiss, MD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Anne Riddell, Jun Pie, Johnny N Mahlangu, MD, Michael Recht, MD PhD, Yu-Min Shen, MD, Kathleen G. Halka, MD, Michael M Meagher, PhD, Arthur W. Nienhuis, MD, Andrew M. Davidoff, MD, Sarah Mangles, Chris L Morton, Zhou Junfang, Vlad Calin Radulescu, MD

Blood (2018) 132 (Supplement 1): 491.

https://doi.org/10.1182/blood-2018-99-118334

Published: 2018

Journal Articles

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

Amit C Nathwani, MD PhD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Doyoung Lee, PhD, Cecilia Rosales, PhD, Mark Phillips, Jun Pie, Zhou Junfang, Michael M Meagher, PhD, Ulrike Reiss, MD, Andrew M. Davidoff, MD, Chris L Morton, Anne Riddell

Blood (2018) 132 (Supplement 1): 489.

https://doi.org/10.1182/blood-2018-99-118256

Published: 2018

Journal Articles

Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice

Azadeh Kia, PhD, Jenny McIntosh, PhD, Cecilia Rosales, PhD, Paniz Hosseini, Rose Sheridan, Justyna Spiewak, Kevin Mills, Romuald Corbau, PhD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 2209.

https://doi.org/10.1182/blood-2018-99-116632

Published: 2018

Journal Articles

Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B

Allison Dane, PhD, Jenny McIntosh, Doyoung Lee, PhD, Rose Sheridan, Mark Maginn, Romuald Corbau, PhD, Andrew M. Davidoff, MD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 2197.

https://doi.org/10.1182/blood-2018-99-117969

Published: 2018

Journal Articles

A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

Pratima Chowdary, MD, Susie Shapiro, MD, Andrew M. Davidoff, MD, Ulrike Reiss, MD, Rebecca Alade, PhD, Geoff Brooks, BSc, Allison Dane, PhD, Jenny McIntosh, PhD, Gerard Short, MBBS, Edward Tuddenham, MD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 631.

https://doi.org/10.1182/blood-2018-99-118050

Published: 2018

Journal Articles

Cytotoxic CD4+ Cells in Chronic Lymphocytic Leukaemia: An Extended Immunophenotypic Analysis Examining Their Association with Cytomegalovirus Serostatus and Similarities with Cytotoxic CD8+ Cells

Maria A V Marzolini, Ayse Akarca, Karen Teng, Teresa Marafioti, MD, Satyen H Gohil, Amit C Nathwani, MD PhD, Evan Newell, PhD, Sergio A Quezada, Karl S Peggs

Blood (2018) 132 (Supplement 1): 3130.

https://doi.org/10.1182/blood-2018-99-116798

Published: 2018

Journal Articles

Adeno-Associated Virus Mediated Gene Transfer with Hyperfunctional FIX-E410H Variant Improves Mean Factor IX Activity Levels in Hemophilia B Mice

Sandra Le Quellec, MD, Allison P Dane, PhD, Claude Negrier, Yesim Dargaud, MD PhD, Amit C Nathwani, MD PhD, Nathalie Enjolras, PhD

Blood (2017) 130 (Supplement 1): 4610.

https://doi.org/10.1182/blood.V130.Suppl_1.4610.4610

Published: 2017

Journal Articles

Novel Humanised ROR1 Chimeric Antigen Receptors for the Treatment of Haematological Malignancies

Satyen Harish Gohil, MBBS MRCP FRCPath, Marco Della Peruta, PhD, Solange R Paredes-Moscosso, Micaela Harrasser, Gordon Weng-Kit Cheung, MSc, David M Davies, PhD, Martin A Pule, MD PhD, Amit C Nathwani, MD PhD

Blood (2016) 128 (22): 3361.

https://doi.org/10.1182/blood.V128.22.3361.3361

Published: 2016

Journal Articles

A Novel Humanised ROR1 Bi-Specific T-Cell Engager Molecule for the Treatment of Chronic Lymphocytic Leukaemia

Satyen Harish Gohil, MBBS MRCP FRCPath, Micaela Harrasser, Solange R Paredes-Moscosso, Amit C Nathwani, MD PhD, Marco Della Peruta, PhD

Blood (2016) 128 (22): 3244.

https://doi.org/10.1182/blood.V128.22.3244.3244

Published: 2016

Journal Articles

Novel ROR1 Antibody Is Able to Trigger Specific and Superior Complement-Dependent Cytotoxicity (CDC) on Primary CLL Cells

Solange R Paredes-Moscosso, Marco Della Peruta, PhD, Satyen Harish Gohil, MBBS MRCP FRCPath, Micaela Harrasser, Amit C Nathwani, MD PhD

Blood (2016) 128 (22): 2052.

https://doi.org/10.1182/blood.V128.22.2052.2052

Published: 2016

Journal Articles

A Novel Human Factor IX Expression Cassette Packaged as Self-Complementary DNA Dimers in Adeno-Associated Virus Vectors Results in Dramatically Improved Liver Transduction, Significantly Improving Prospects for Hemophilia B Gene Therapy.

John T. Gray, PhD, Andrew M. Davidoff, MD, Amit C. Nathwani, MD, PhD

Blood (2004) 104 (11): 3181.

https://doi.org/10.1182/blood.V104.11.3181.3181

Published: 2004

NARROW

Evaluating ROR1 As a Target in Childhood Leukemia

Liver-Directed AAV Gene Therapy for Gaucher Disease

Enhancing FVIII Expression By Modifying B-Domain Sequence for Hemophilia A Gene Therapy

A Novel Lysine to Arginine Substitution at Position 301 Enhances Activity of Factor IX

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice

Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B

A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

Cytotoxic CD4+ Cells in Chronic Lymphocytic Leukaemia: An Extended Immunophenotypic Analysis Examining Their Association with Cytomegalovirus Serostatus and Similarities with Cytotoxic CD8+ Cells

Adeno-Associated Virus Mediated Gene Transfer with Hyperfunctional FIX-E410H Variant Improves Mean Factor IX Activity Levels in Hemophilia B Mice

Novel Humanised ROR1 Chimeric Antigen Receptors for the Treatment of Haematological Malignancies

A Novel Humanised ROR1 Bi-Specific T-Cell Engager Molecule for the Treatment of Chronic Lymphocytic Leukaemia

Novel ROR1 Antibody Is Able to Trigger Specific and Superior Complement-Dependent Cytotoxicity (CDC) on Primary CLL Cells

A Novel Human Factor IX Expression Cassette Packaged as Self-Complementary DNA Dimers in Adeno-Associated Virus Vectors Results in Dramatically Improved Liver Transduction, Significantly Improving Prospects for Hemophilia B Gene Therapy.

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ASH Publications

American Society of Hematology

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ASH Publications

American Society of Hematology

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