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Journal Articles

A Synthetic Receptor Enables Breakthrough Scale Manufacturing of iPSC-Derived CD19-CAR Cell Therapies for Hematologic and Autoimmune Diseases

Dillon Jarrell, David Vereide, Cassidy Arnold, Max Murphey, Ashley Yingst, Ryan Koning, Nikole Perdue, Artem Romanov, Celeste Busch, Igor Slukvin, Ryan Crisman, Ryan P. Larson, Andrew M. Scharenberg, Branden Salinas, Samantha Ohara

Blood (2024) 144 (Supplement 1): 148.

https://doi.org/10.1182/blood-2024-200130

Published: 2024

Journal Articles

Vivovec™ Surface-Engineered Lentiviral Particles Mediate In Vivo CAR T Generation with Potent and Highly Durable Activity in Non-Human Primates

Blood (2024) 144 (Supplement 1): 502.

https://doi.org/10.1182/blood-2024-206328

Published: 2024

Journal Articles

Nonclinical Toxicology, Biodistribution, and Pharmacokinetics of UB-VV111, an In Vivo Anti-CD19 CAR T Cell Therapy

Alissa H. Brandes, Angela Soo Min Koo, Anai Perez, Kathryn Michels, Travis Friesen, Alyssa Sheih, Don Parilla, Susana Hernandez, Nolan Ericson, Sarah Dickerson, Mason Muhonen, Jessica Freeman, Sarah Gould, Alessandra Maria Sullivan, Jeffrey Teoh, Byoung Y. Ryu, Andrew M. Scharenberg, Ryan P. Larson

Blood (2024) 144 (Supplement 1): 2046.

https://doi.org/10.1182/blood-2024-211032

Published: 2024

Journal Articles

In vivo CAR T-cell generation in nonhuman primates using lentiviral vectors displaying a multidomain fusion ligand

Blood (2024) 144 (9): 977–987.

https://doi.org/10.1182/blood.2024024523

Published: 2024

Includes: Supplemental data

Journal Articles

Vivovec™ Surface-Engineered Lentiviral Particles Mediate In Vivo CAR T Generation with Potent and Highly Durable Activity in Non-Human Primates and Tumor-Bearing Humanized Mice

Maura Parker, Justin Ulrich-Lewis, Weiliang Tang, Chris Nicolai, Kathryn Michels, Susana Hernandez, Don Parrilla, Sara Cooper, Anai Perez, Mollie McDonnell, Greyson Hamilton, Chang-Chih Wu, Seungjin Shin, Andrew M. Scharenberg, Ryan P. Larson, Laurie Beitz, Byoung Y. Ryu

Blood (2023) 142 (Supplement 1): 765.

https://doi.org/10.1182/blood-2023-182195

Published: 2023

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

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

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

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

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

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

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

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

Ethical and regulatory aspects of genome editing

Donald B. Kohn, Matthew H. Porteus, Andrew M. Scharenberg

Blood (2016) 127 (21): 2553–2560.

https://doi.org/10.1182/blood-2016-01-678136

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

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

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

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

Blood (2014) 124 (21): SCI-11.

https://doi.org/10.1182/blood.V124.21.SCI-11.SCI-11

Published: 2014

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

NARROW

A Synthetic Receptor Enables Breakthrough Scale Manufacturing of iPSC-Derived CD19-CAR Cell Therapies for Hematologic and Autoimmune Diseases

Vivovec™ Surface-Engineered Lentiviral Particles Mediate In Vivo CAR T Generation with Potent and Highly Durable Activity in Non-Human Primates

Nonclinical Toxicology, Biodistribution, and Pharmacokinetics of UB-VV111, an In Vivo Anti-CD19 CAR T Cell Therapy

In vivo CAR T-cell generation in nonhuman primates using lentiviral vectors displaying a multidomain fusion ligand

Vivovec™ Surface-Engineered Lentiviral Particles Mediate In Vivo CAR T Generation with Potent and Highly Durable Activity in Non-Human Primates and Tumor-Bearing Humanized Mice

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

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

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

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

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

Talen Mediated Therapeutic Gene Editing Strategy for ß-Hemoglobinopathies

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

Ethical and regulatory aspects of genome editing

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

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

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

Genome Editing Toolbox

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

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American Society of Hematology

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American Society of Hematology

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