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David E. Paschon
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Journal Articles

ZFN-Mediated Gene Targeting at the Albumin Locus in Liver Results in Therapeutic Levels of Human FIX in Mice and Non-Human Primates

Thomas Wechsler, PhD, Kathleen E. Meyer, S. Kaye Spratt, Judith Greengard, PhD, Yolanda Santiago, Scott Sproul, Richard Surosky, David E. Paschon, Nathalie Dubois-Stringfellow, Dale Genji Ando, MD, Geoff Nichol, Edward J. Rebar, PhD, Michael C. Holmes, PhD
Journal: Blood
Blood (2015) 126 (23): 200.
https://doi.org/10.1182/blood.V126.23.200.200
Published: 2015
Journal Articles

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Rajiv Sharma, Xavier M. Anguela, Yannick Doyon, Thomas Wechsler, Russell C. DeKelver, Scott Sproul, David E. Paschon, Jeffrey C. Miller, Robert J. Davidson, David Shivak, Shangzhen Zhou, Julianne Rieders, Philip D. Gregory, Michael C. Holmes, Edward J. Rebar, Katherine A. High
Journal: Blood
Blood (2015) 126 (15): 1777–1784.
https://doi.org/10.1182/blood-2014-12-615492
Published: 2015
Includes: Supplemental data
Journal Articles

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

Megan D. Hoban, Gregory J. Cost, Matthew C. Mendel, Zulema Romero, Michael L. Kaufman, Alok V. Joglekar, Michelle Ho, Dianne Lumaquin, David Gray, Georgia R. Lill, Aaron R. Cooper, Fabrizia Urbinati, Shantha Senadheera, Allen Zhu, Pei-Qi Liu, David E. Paschon, Lei Zhang, Edward J. Rebar, Andrew Wilber, Xiaoyan Wang, Philip D. Gregory, Michael C. Holmes, Andreas Reik, Roger P. Hollis, Donald B. Kohn
Journal: Blood
Blood (2015) 125 (17): 2597–2604.
https://doi.org/10.1182/blood-2014-12-615948
Published: 2015
Includes: Supplemental data
Journal Articles

HPRT As a Selectable Safe Harbor for Transgenesis

Anthony Conway, PhD, Josée Laganière, PhD, David E Paschon, PhD, Katrin Hacke, PhD, Noriyuki Kasahara, MD PhD, Philip D Gregory, PhD, Michael C. Holmes, PhD, Gregory J Cost, PhD
Journal: Blood
Blood (2014) 124 (21): 4796.
https://doi.org/10.1182/blood.V124.21.4796.4796
Published: 2014
Journal Articles

Targeted Gene Modification In Hematopoietic Stem Cells: A Potential Treatment For Thalassemia and Sickle Cell Anemia

Andreas Reik, PhD, Kai-Hsin Chang, PhD, Sandra Stehling-Sun, PhD, Yuanyue Zhou, Gary K Lee, PhD, Lynn Truong, Travis Wood, Ziying Zhang, Albert Luong, Andy Chan, Pei-Qi Liu, PhD, Jeffrey C Miller, PhD, David E Paschon, PhD, Dmitry Y Guschin, PhD, Lei Zhang, PhD, Evangelia Yannaki, MD, Martin A Giedlin, PhD, Edward J Rebar, PhD, Philip D Gregory, PhD, Fyodor D Urnov, PHD, Thalia Papayannopoulou, MD, George Stamatoyannopoulos, MD, DSci
Journal: Blood
Blood (2013) 122 (21): 434.
https://doi.org/10.1182/blood.V122.21.434.434
Published: 2013
Journal Articles

ZFN Mediated Targeting Of Albumin “Safe Harbor” Results In Therapeutic Levels Of Human Factor VIII In a Mouse Model Of Hemophilia A

Xavier M Anguela, PhD, Rajiv Sharma, Yannick Doyon, PhD, Thomas Wechsler, PhD, David E Paschon, PhD, Robert J Davidson, Shangzhen Zhou, MD, Philip D Gregory, PhD, Michael C. Holmes, PhD, Edward J Rebar, PhD, Katherine A. High, MD
Journal: Blood
Blood (2013) 122 (21): 720.
https://doi.org/10.1182/blood.V122.21.720.720
Published: 2013
Journal Articles

In Vivo Genome Editing of Liver Albumin for Therapeutic Gene Expression: Rescue of Hemophilic Mice Via Integration of Factor 9

Xavier M Anguela, PhD, Rajiv Sharma, Yannick Doyon, PhD, Sunnie Y Wong, David E Paschon, Hojun Li, MD PhD, Virginia Haurigot, PhD, Robert J Davidson, Shangzhen Zhou, MD, Philip D. Gregory, PhD, Michael C. Holmes, PhD, Edward Rebar, PhD, Katherine A. High, MD
Journal: Blood
Blood (2012) 120 (21): 751.
https://doi.org/10.1182/blood.V120.21.751.751
Published: 2012
Journal Articles

TCR Gene Editing Results in Effective Immunotherapy of Leukemia without the Development of GvHD

Elena Provasi, PhD, Pietro Genovese, Angelo Lombardo, PhD, Zulma Magnani, Pei-Qi Liu, Andreas Reik, Victoria Chu, David E Paschon, Lei Zhang, Jurgen Kuball, MD, PhD, Barbara Camisa, Attilio Bondanza, MD, PhD, Giulia Casorati, PhD, Maurilio Ponzoni, MD, Fabio Ciceri, MD, Claudio Bordignon, MD, Philip D. Greenberg, MD, Michael C. Holmes, PhD, Philip D. Gregory, PhD, Luigi Naldini, MD, PhD, Chiara Bonini, MD
Journal: Blood
Blood (2011) 118 (21): 667.
https://doi.org/10.1182/blood.V118.21.667.667
Published: 2011