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Susannah I. Thornhill
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Journal Articles

Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

Sung-Yun Pai, MD, Luigi Daniele Notarangelo, MD, Chad Harris, Federica Cattaneo, MD, Matthew Wladkowski, Myriam Armant, PhD, Grace S. Kao, MD, Matthew Wielgosz, Arthur W. Nienhuis, MD, Ute Modlich, DVM, PhD, Martijn Brugman, PhD, Axel Schambach, MD PhD, Michael Rothe, Susannah I Thornhill, PhD, H. Bobby Gaspar, Kathryn Parsley, PhD, Samantha Cooray, PhD, Elke Grassman, Ph.D., Frederic Bushman, PhD, Sally Shupien, Matias Oleastro, MD, Salima Hacein-Bey-Abina, Alain Fischer, MD, Marina Cavazzana-Calvo, MD, PhD, Punam Malik, MD, Alexandra Filipovich, MD, Donald B. Kohn, MD, Christopher Baum, MD, David A. Williams, MD, Adrian J Thrasher, MD, PhD
Journal: Blood
Blood (2011) 118 (21): 164.
https://doi.org/10.1182/blood.V118.21.164.164
Published: 2011
Journal Articles

Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells

Fang Zhang, Susannah I. Thornhill, Steven J. Howe, Meera Ulaganathan, Axel Schambach, Joanna Sinclair, Christine Kinnon, H. Bobby Gaspar, Michael Antoniou, Adrian J. Thrasher
Journal: Blood
Blood (2007) 110 (5): 1448–1457.
https://doi.org/10.1182/blood-2006-12-060814
Published: 2007
Includes: Supplemental data