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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

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

Blood (2019) 134 (Supplement_1): 3355.

https://doi.org/10.1182/blood-2019-130945

Published: 2019

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

Blood (2018) 132 (Supplement 1): 3490.

https://doi.org/10.1182/blood-2018-99-117456

Published: 2018

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

Blood (2018) 132 (Supplement 1): 806.

https://doi.org/10.1182/blood-2018-99-112996

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

Blood (2017) 130 (Supplement 1): 3340.

https://doi.org/10.1182/blood.V130.Suppl_1.3340.3340

Published: 2017

Journal Articles

Using Multiplexed Genome Editing to Distinguish between Loss- and Gain-of-Function Variants in CARD11

Richard James, PhD, Iana Meitlis, BS, King L Hung, BS, David J. Rawlings, MD, Eric J Allenspach, MD PhD, Lea M Starita, PhD

Blood (2017) 130 (Supplement 1): 622.

https://doi.org/10.1182/blood.V130.Suppl_1.622.622

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

Blood (2016) 128 (22): 1348.

https://doi.org/10.1182/blood.V128.22.1348.1348

Published: 2016

Journal Articles

High Titer Lentivector from a Stable Lenti-Producer Efficiently Corrects CD34+ Hematopoietic Stem Cells from Patients with p47phox-Deficient Chronic Granulomatous Disease

Uimook Choi, PhD, Narda Theobald, Throm E Robert, PhD, John Gray, PhD, David J. Rawlings, MD, Brian P. Sorrentino, MD, Harry Malech, Suk See De Ravin, MD

Blood (2015) 126 (23): 2036.

https://doi.org/10.1182/blood.V126.23.2036.2036

Published: 2015

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

Blood (2015) 126 (23): 262.

https://doi.org/10.1182/blood.V126.23.262.262

Published: 2015

Journal Articles

Immunomodulation of Transgene Responses Following Naked DNA Transfer of Factor VIII into Hemophilia A Mice.

Peiqing Ye, MD, David J. Rawlings, MD, Arthur R. Thompson, MD, PhD, Hans D. Ochs, MD,Dr.Med, Carol H. Miao, PhD

Blood (2005) 106 (11): 1279.

https://doi.org/10.1182/blood.V106.11.1279.1279

Published: 2005

NARROW

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

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

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

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

Talen Mediated Therapeutic Gene Editing Strategy for ß-Hemoglobinopathies

Using Multiplexed Genome Editing to Distinguish between Loss- and Gain-of-Function Variants in CARD11

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

High Titer Lentivector from a Stable Lenti-Producer Efficiently Corrects CD34+ Hematopoietic Stem Cells from Patients with p47phox-Deficient Chronic Granulomatous Disease

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

Immunomodulation of Transgene Responses Following Naked DNA Transfer of Factor VIII into Hemophilia A Mice.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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