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Andrew M. Scharenberg, MD
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Journal Articles

Enabling Gene-Edited, Regulatory-like, T Cells (edTreg) for Treatment of IPEX and Other Autoimmune Disorders

Yuchi Honaker, PhD, Karen Sommer, PhD, Noelle Dahl, Yufei Xiang, MD, PhD, Christina Lopez, PhD, Samuel Scharffenberger, Michelle Christian, Troy R. Torgerson, MD PhD, Andrew M. Scharenberg, MD, David J. Rawlings, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2071.
https://doi.org/10.1182/blood-2019-131946
Published: 2019
Journal Articles

Manufacture and In Vivo Support of Gene-Edited, Regulatory-like, T-Cells (edTreg) Using a Synthetic IL2 Receptor

Gene I. Uenishi, PhD, Cornell Mallari, Luis Gamboa, Samuel West, Chester Jacobs, Noelle Dahl, Greg Boucher, Samantha Chin, Rangoli Aeran, Dariusz Wodziak, Simon Gebremeskel, Karen Vo, PhD, Cade E. Ito, Laura Smith, Chandra Patel, PhD, Keith Abe, PhD, Basha Stankovich, PhD, Karen Sommer, PhD, Troy R. Torgerson, MD PhD, David J. Rawlings, MD, Andrew M. Scharenberg, MD, Greg J. Cost, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3355.
https://doi.org/10.1182/blood-2019-130945
Published: 2019
Journal Articles

Persistence of CRISPR/Cas9-Edited Hematopoietic Stem and Progenitor Cells and Reactivation of Fetal Hemoglobin in Nonhuman Primates

Olivier Humbert, PhD, Stefan Radtke, PhD, Ray R Carillo, Anai M Perez, Sowmya Somashekar Reddy, Lauren E Schefter, Christopher Lux, MDPhD, Sowmya Pattabhi, PhD, Olivier Negre, PhD, Ciaran M Lee, PhD, Jennifer Adair, PhD, Christopher Peterson, PhD, Gang Bao, PhD, David J. Rawlings, MD, Andrew M. Scharenberg, MD, Hans-Peter Kiem, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 806.
https://doi.org/10.1182/blood-2018-99-112996
Published: 2018
Journal Articles

Genome Editing Via NHEJ-Mediated Targeted Integration Retains Engraftment Potential of Edited Hematopoietic Stem Cells

Gene I. Uenishi, PhD, Keith Abe, Bee-Chun Sun, Cornell Mallari, Andrew M. Scharenberg, MD, Gregory J. Cost, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3480.
https://doi.org/10.1182/blood-2018-99-117488
Published: 2018
Journal Articles

Conversion of T-Effector Cells to Immunosuppressive T-Regulatory-like Cells By CRISPR/Cas9-Mediated Integration of a FOXP3 Transgene

Yuchi Honaker, PhD, Yufei Xiang, MD, PhD, Logan Fisher, B.S., Karen Sommer, PhD, Troy R. Torgerson, MD PhD, Andrew M. Scharenberg, MD, David J. Rawlings, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3490.
https://doi.org/10.1182/blood-2018-99-117456
Published: 2018
Journal Articles

Chemically Controlled, Immunosuppression-Resistant, Anti-Bcma CAR-T Cells for Treatment of Antibody-Mediated Autoimmunity

Sowndharya Rajavel, Cade E. Ito, Keith Abe, Valerie Guerrero, Gene I. Uenishi, PhD, Andrew M. Scharenberg, MD, Gregory J. Cost, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 2203.
https://doi.org/10.1182/blood-2018-99-117630
Published: 2018
Journal Articles

Talen Mediated Therapeutic Gene Editing Strategy for ß-Hemoglobinopathies

Christopher Lux, MDPhD, Kyle Jacoby, PhD, Sowmya Pattabhi, PhD, Julia Yang, BS, Olivier Humbert, PhD, Brian Gordon, MS, Irwin D. Bernstein, MD, David J. Rawlings, MD, Andrew M. Scharenberg, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 3340.
https://doi.org/10.1182/blood.V130.Suppl_1.3340.3340
Published: 2017
Journal Articles

Rapid Expansion of Gene-Marked Lymphocytes in X-SCID Dogs after AMD3100+G-CSF-Based Hematopoietic Stem/Progenitor Cell Mobilization and Intravenous Injection of a Common γ-Chain Expressing Foamy Viral Vector

Frieda Chan, PhD, Olivier Humbert, PhD, Troy Torgerson, MD PhD, Nicholas Hubbard, Patricia O'Donnell, Daniel R Humphrys, B.S., Jennifer E Adair, PhD, Peter Felsburg, Grant D. Trobridge, PhD, Andrew M. Scharenberg, MD, David J. Rawlings, MD, Hans-Peter Kiem, MD FACP
Journal: Blood
Blood (2016) 128 (22): 1348.
https://doi.org/10.1182/blood.V128.22.1348.1348
Published: 2016
Journal Articles

Pgk-Mediated Expression of Common Gamma Chain Is More Effective Than EF1a for Therapeutic Immune Reconstitution of X-SCID Dogs after In Vivo Gene Therapy with Foamy Virus Vector

Olivier Humbert, PhD, Christopher R Burtner, PhD, Patricia O'Donnell, Daniel R Humphrys, B.S., Nicholas Hubbard, Frieda Chan, PhD, Jennifer E Adair, PhD, Grant D. Trobridge, PhD, Troy Torgerson, MD PhD, Andrew M. Scharenberg, MD, David J. Rawlings, MD, Peter Felsburg, Hans-Peter Kiem, MD
Journal: Blood
Blood (2015) 126 (23): 262.
https://doi.org/10.1182/blood.V126.23.262.262
Published: 2015
Journal Articles

Megatal, Crispr/Cas9, and Talen T-Cell Receptor Gene Editing

Mark J. Osborn, PhD, Beau R Webber, PhD, Friederike Knipping, Cara-lin Lonetree, Anthony DeFeo, Amber McElroy, Colby Starker, PhD, Richard Gabriel, Sarah Merkel, Keli L. Hippen, PhD, Daniel Voytas, PhD, Christof von Kalle, MD, Manfred Schmidt, PhD, Jeffrey S. Miller, MD, Jordan Jarjour, PhD, Alexander Astrakhan, PhD, Andrew M Scharenberg, MD, Jakub Tolar, MD, PhD, Bruce R. Blazar, MD
Journal: Blood
Blood (2015) 126 (23): 2045.
https://doi.org/10.1182/blood.V126.23.2045.2045
Published: 2015
Journal Articles

Robust Therapeutic Expression of the Common Gamma Chain with the Human Pgk Promoter Using Foamy Virus in Vivo Gene Therapy in a Canine Model of Severe Combined Immunodeficiency

Christopher R Burtner, PhD, Olivier Humbert, PhD, Patricia O'Donnell, Nicholas Hubbard, Daniel Humphrys, Jennifer E Adair, PhD, Grant D Trobridge, PhD, Troy R Torgerson, MD PhD, Andrew M Scharenberg, MD, David Rawlings, MD, Peter J Felsburg, VMD, PhD, Hans-Peter Kiem, MD
Journal: Blood
Blood (2014) 124 (21): 4794.
https://doi.org/10.1182/blood.V124.21.4794.4794
Published: 2014
Journal Articles

Genome Editing Toolbox

Andrew M. Scharenberg, MD
Journal: Blood
Blood (2014) 124 (21): SCI-11.
https://doi.org/10.1182/blood.V124.21.SCI-11.SCI-11
Published: 2014