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Cecilia Rosales
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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
Journal: Blood
Blood (2018) 132 (Supplement 1): 3484.
https://doi.org/10.1182/blood-2018-99-113830
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
Journal: Blood
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
Journal: Blood
Blood (2018) 132 (Supplement 1): 2209.
https://doi.org/10.1182/blood-2018-99-116632
Published: 2018
Journal Articles

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

Jenny McIntosh, Peter J. Lenting, Cecilia Rosales, Doyoung Lee, Samira Rabbanian, Deepak Raj, Nishil Patel, Edward G. D. Tuddenham, Olivier D. Christophe, John H. McVey, Simon Waddington, Arthur W. Nienhuis, John T. Gray, Paolo Fagone, Federico Mingozzi, Shang-Zhen Zhou, Katherine A. High, Maria Cancio, Catherine Y. C. Ng, Junfang Zhou, Christopher L. Morton, Andrew M. Davidoff, Amit C. Nathwani
Journal: Blood
Blood (2013) 121 (17): 3335–3344.
https://doi.org/10.1182/blood-2012-10-462200
Published: 2013
Includes: Supplemental data
Journal Articles

Stable Factor IX Activity Following AAV-Mediated Gene Transfer in Patients with Severe Hemophilia B

Andrew Davidoff, MD, Edward GD Tuddenham, MBBS, FRCP, FRCPath, PhD, Savita Rangarajan, MBBS, FRCP, FRCPath, Cecilia Rosales, PhD, Jenny McIntosh, PhD, Pratima Chowdary, MBBS, FRCP, FRCPath, Anne Riddell, BSc, Bertil Glader, MD, PhD, Pradip Rustagi, MD, Catherine Ng, MS, Mark Kay, MD, PhD, Junfang Zhou, MD, Yunyu Spence, PhD, Christopher Morton, B.S., James Allay, PhD, Susan Sleep, PhD, Etiena Basner-Tschakarjan, MD, Federico Mingozzi, PhD., Katherine A. High, MD, John T. Gray, PhD, Ulrike M. Reiss, MD, Arthur W. Nienhuis, MD, Amit C Nathwani, MBChB, FRCP, FRCPath, PhD
Journal: Blood
Blood (2012) 120 (21): 752.
https://doi.org/10.1182/blood.V120.21.752.752
Published: 2012
Journal Articles

Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B

Amit C Nathwani, MBChB, FRCP, FRCPath, PhD, Edward GD Tuddenham, MBBS, FRCP, FRCPath, PhD, Savita Rangarajan, MBBS, FRCP, FRCPath, Cecilia Rosales, PhD, Jenny H McIntosh, PhD, David C Linch, MD FMedSci, Pratima Chowdary, MBBS, FRCP, FRCPath, Anja Griffioen, Anne Riddell, BSc, Jun Pie, BSN, Chris Harrington, BSN, James O’Beirne, MBBS, FRCP, MD EDIC, Keith Smith, BSc, MSc, C.Biol, John Pasi, MBChB, PhD, FRCP, FRCPath, Bertil Glader, MD PhD, Pradip Rustagi, MD, Catherine Ng, MS, Mark Kay, MD, PhD, Junfang Zhou, MD, Yunyu Spence, PhD, Christopher Morton, BS, James Allay, PhD, John Coleman, Susan Sleep, PhD, John M Cunningham, MD, Etiena Basner-Tschakarjan, MD, Federico Mingozzi, PhD, Katherine A. High, MD, John T. Gray, PhD, Ulrike M. Reiss, MD, Arthur W. Nienhuis, MD, Andrew Davidoff, MD
Journal: Blood
Blood (2011) 118 (21): 5.
https://doi.org/10.1182/blood.V118.21.5.5
Published: 2011
Journal Articles

Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B

Amit Nathwani, MD, PhD, Edward Tuddenham, MD, Cecilia Rosales, Jenny McIntosh, Anne Riddell, Pradip Rustagi, Bertil Glader, Mark Kay, MD, PhD, James Allay, PhD, John Coleman, Susan Sleep, Katherine A. High, MD, Federico Mingozzi, PhD., John T. Gray, PhD, Ulrike M. Reiss, MD, Arthur W. Nienhuis, MD, Andrew Davidoff
Journal: Blood
Blood (2010) 116 (21): 248.
https://doi.org/10.1182/blood.V116.21.248.248
Published: 2010