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Journal Articles

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Lauri M. Burroughs, Aleksandra Petrovic, Ruta Brazauskas, Xuerong Liu, Linda M. Griffith, Hans D. Ochs, Jack J. Bleesing, Stephanie Edwards, Christopher C. Dvorak, Sonali Chaudhury, Susan E. Prockop, Ralph Quinones, Frederick D. Goldman, Troy C. Quigg, Shanmuganathan Chandrakasan, Angela R. Smith, Suhag Parikh, Blachy J. Dávila Saldaña, Monica S. Thakar, Rachel Phelan, Shalini Shenoy, Lisa R. Forbes, Caridad Martinez, Deepak Chellapandian, Evan Shereck, Holly K. Miller, Neena Kapoor, Jessie L. Barnum, Hey Chong, David C. Shyr, Karin Chen, Rolla Abu-Arja, Ami J. Shah, Katja G. Weinacht, Theodore B. Moore, Avni Joshi, Kenneth B. DeSantes, Alfred P. Gillio, Geoffrey D. E. Cuvelier, Michael D. Keller, Jacob Rozmus, Troy Torgerson, Michael A. Pulsipher, Elie Haddad, Kathleen E. Sullivan, Brent R. Logan, Donald B. Kohn, Jennifer M. Puck, Luigi D. Notarangelo, Sung-Yun Pai, David J. Rawlings, Morton J. Cowan

Blood (2020) 135 (23): 2094–2105.

https://doi.org/10.1182/blood.2019002939

Published: 2020

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

Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome

Nicholas Hubbard, David Hagin, Karen Sommer, Yumei Song, Iram Khan, Courtnee Clough, Hans D. Ochs, David J. Rawlings, Andrew M. Scharenberg, Troy R. Torgerson

Blood (2016) 127 (21): 2513–2522.

https://doi.org/10.1182/blood-2015-11-683235

Published: 2016

Includes: Supplemental data

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

Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells

Cathy X. Wang, Blythe D. Sather, Xuefeng Wang, Jennifer Adair, Iram Khan, Swati Singh, Shanshan Lang, Amie Adams, Gabrielle Curinga, Hans-Peter Kiem, Carol H. Miao, David J. Rawlings, Bruce E. Torbett

Blood (2014) 124 (6): 913–923.

https://doi.org/10.1182/blood-2013-12-546218

Published: 2014

Includes: Supplemental data

Journal Articles

Intravenous injection of a foamy virus vector to correct canine SCID-X1

Christopher R. Burtner, Brian C. Beard, Douglas R. Kennedy, Martin E. Wohlfahrt, Jennifer E. Adair, Grant D. Trobridge, Andrew M. Scharenberg, Troy R. Torgerson, David J. Rawlings, Peter J. Felsburg, Hans-Peter Kiem

Blood (2014) 123 (23): 3578–3584.

https://doi.org/10.1182/blood-2013-11-538926

Published: 2014

Includes: Supplemental data

Journal Articles

Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome

Alexander Astrakhan, Blythe D. Sather, Byoung Y. Ryu, Socheath Khim, Swati Singh, Stephanie Humblet-Baron, Hans D. Ochs, Carol H. Miao, David J. Rawlings

Blood (2012) 119 (19): 4395–4407.

https://doi.org/10.1182/blood-2011-03-340711

Published: 2012

Includes: Supplemental data

Journal Articles

B cell–specific lentiviral gene therapy leads to sustained B-cell functional recovery in a murine model of X-linked agammaglobulinemia

Hannah M. Kerns, Byoung Y. Ryu, Brigid V. Stirling, Blythe D. Sather, Alexander Astrakhan, Stephanie Humblet-Baron, Denny Liggitt, David J. Rawlings

Blood (2010) 115 (11): 2146–2155.

https://doi.org/10.1182/blood-2009-09-241869

Published: 2010

Includes: Supplemental data

Journal Articles

Anti-CD3 antibodies modulate anti–factor VIII immune responses in hemophilia A mice after factor VIII plasmid-mediated gene therapy

Baowei Peng, Peiqing Ye, David J. Rawlings, Hans D. Ochs, Carol H. Miao

Blood (2009) 114 (20): 4373–4382.

https://doi.org/10.1182/blood-2009-05-217315

Published: 2009

Includes: Supplemental data

Journal Articles

CD4 + FOXP3 + regulatory T cells confer long-term regulation of factor VIII–specific immune responses in plasmid-mediated gene therapy–treated hemophilia mice

Carol H. Miao, Benjamin R. Harmeling, Steven F. Ziegler, Benjamin C. Yen, Troy Torgerson, Liping Chen, Roger J. Yau, Baowei Peng, Arthur R. Thompson, Hans D. Ochs, David J. Rawlings

Blood (2009) 114 (19): 4034–4044.

https://doi.org/10.1182/blood-2009-06-228155

Published: 2009

Includes: Supplemental data

Journal Articles

Wiskott-Aldrich syndrome protein deficiency in B cells results in impaired peripheral homeostasis

Almut Meyer-Bahlburg, Shirly Becker-Herman, Stephanie Humblet-Baron, Socheath Khim, Michele Weber, Gerben Bouma, Adrian J. Thrasher, Facundo D. Batista, David J. Rawlings

Blood (2008) 112 (10): 4158–4169.

https://doi.org/10.1182/blood-2008-02-140814

Published: 2008

Includes: Supplemental data

Journal Articles

Transient blockade of the inducible costimulator pathway generates long-term tolerance to factor VIII after nonviral gene transfer into hemophilia A mice

Baowei Peng, Peiqing Ye, Bruce R. Blazar, Gordon J. Freeman, David J. Rawlings, Hans D. Ochs, Carol H. Miao

Blood (2008) 112 (5): 1662–1672.

https://doi.org/10.1182/blood-2008-01-128413

Published: 2008

Journal Articles

Immunomodulation of transgene responses following naked DNA transfer of human factor VIII into hemophilia A mice

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

Blood (2006) 108 (1): 19–27.

https://doi.org/10.1182/blood-2005-11-4532

Published: 2006

NARROW

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

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

Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome

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

Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells

Intravenous injection of a foamy virus vector to correct canine SCID-X1

Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome

B cell–specific lentiviral gene therapy leads to sustained B-cell functional recovery in a murine model of X-linked agammaglobulinemia

Anti-CD3 antibodies modulate anti–factor VIII immune responses in hemophilia A mice after factor VIII plasmid-mediated gene therapy

CD4 + FOXP3 + regulatory T cells confer long-term regulation of factor VIII–specific immune responses in plasmid-mediated gene therapy–treated hemophilia mice

Wiskott-Aldrich syndrome protein deficiency in B cells results in impaired peripheral homeostasis

Transient blockade of the inducible costimulator pathway generates long-term tolerance to factor VIII after nonviral gene transfer into hemophilia A mice

Immunomodulation of transgene responses following naked DNA transfer of human 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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