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Journal Articles

Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector

Blood (2013) 122 (21): 715.

https://doi.org/10.1182/blood.V122.21.715.715

Published: 2013

Journal Articles

Overexpression of EVI1 Causes Genomic Instability and Cell Cycle Arrest in Hematopoietic Cells.

Friederike Herbst, PhD, Claudia R Ball, PhD, Oksana Zavidij, PhD, Annika Mengering, Sylvia Fessler, Manfred Schmidt, PhD, Christof von Kalle, MD, PhD, Hanno Glimm, MD

Blood (2012) 120 (21): 2398.

https://doi.org/10.1182/blood.V120.21.2398.2398

Published: 2012

Journal Articles

Lentiviral Vector Induced Insertional Haploinsufficiency of Ebf1 Causes Leukemia in a Murine Bone Marrow Transplantation Model

Dirk Heckl, PhD, Adrian Schwarzer, MD, Reinhard Haemmerle, Johann Meyer, PhD, Doris Steinemann, PhD, Cornelia Rudolph, PhD, Britta Skawran, PhD, Brigitte Schlegelberger, MD, Christopher Baum, MD, Ute Modlich, DVM, PhD

Blood (2011) 118 (21): 671.

https://doi.org/10.1182/blood.V118.21.671.671

Published: 2011

Journal Articles

Deregulated EVI1 Expression Leads to Genomic Instability and G1 Cell Cycle Arrest

Friederike Herbst, Claudia R Ball, PhD, Wei Wang, Kadin Karakaya, Sylvia Fessler, Alwin Krämer, MD, Manfred Schmidt, PhD, Christof von Kalle, MD, Hanno Glimm, MD

Blood (2011) 118 (21): 2431.

https://doi.org/10.1182/blood.V118.21.2431.2431

Published: 2011

Journal Articles

Integration-Mediated Activation of PRDM16 and HMGA2 in Multiple Clones without Adverse Hematopoietic Consequences Following Transplant of Autologous MGMTP140K Gene-Modified CD34 + Cells

Jennifer E Adair, PhD, Brian C Beard, PhD, Grant Trobridge, PhD, Frederic Bushman, PhD, Maciej M Mrugala, MD PhD, Hans-Peter Kiem, MD

Blood (2011) 118 (21): 2053.

https://doi.org/10.1182/blood.V118.21.2053.2053

Published: 2011

Journal Articles

Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

Blood (2011) 118 (21): 164.

https://doi.org/10.1182/blood.V118.21.164.164

Published: 2011

Journal Articles

Notch Expression Expands the Pre-Malignant Pool of T-Cell Acute Lymphoblastic Leukemia Clones without Affecting Self-Renewal

Jessica S Blackburn, PhD, Sali Liu, BS, David M. Langenau, PhD

Blood (2010) 116 (21): 3161.

https://doi.org/10.1182/blood.V116.21.3161.3161

Published: 2010

Journal Articles

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

Gary P. Wang, Charles C. Berry, Nirav Malani, Philippe Leboulch, Alain Fischer, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo, Frederic D. Bushman

Blood (2010) 115 (22): 4356–4366.

https://doi.org/10.1182/blood-2009-12-257352

Published: 2010

Includes: Supplemental data

Journal Articles

Analysis of Viral Integration Sites in Human Induced Pluripotent Stem Cells.

Thomas Winkler, MD, Amy R Cantelina, Jean-Yves Metais, Ph.D., Xiuli Xu, Ph.D., Anh-Dao Nguyen, Ph.D., Tyra Wolfsberg, Ph.D., Jessica Antosiewicz-Bourget, MBA, James Alexander Thomson, Ph.D., Cynthia E. Dunbar, MD

Blood (2009) 114 (22): 1485.

https://doi.org/10.1182/blood.V114.22.1485.1485

Published: 2009

Journal Articles

Pre-Transplant Integration Site Diagnostics in Hematopoietic Stem Cell Gene Transfer.

Claudia R Ball, Sylvia Fessler, Daniela Belle, Manfred Schmidt, Christof von Kalle, Hanno Glimm

Blood (2009) 114 (22): 3581.

https://doi.org/10.1182/blood.V114.22.3581.3581

Published: 2009

Journal Articles

Identification of a TFIID Binding Sequence That Increases Expression from the Ankyrin-1 Promoter: Application to Globin Gene Therapy

Karina Laflamme, PhD, Laura E. Elnitski, PhD, Clara Wong, Yang Mary, PhD, Patrick G. Gallagher, MD, David M. Bodine, PhD

Blood (2007) 110 (11): 3741.

https://doi.org/10.1182/blood.V110.11.3741.3741

Published: 2007

Journal Articles

Reduced Genotoxic Risk Using Foamy Viral Vectors for the Treatment of Canine Leukocyte Adhesion Deficiency

Thomas R. Bauer, Jr, PhD, Mehreen Hai, Ph.D., Rima L. Adler, Ph.D., James M. Allen, Ph.D., Laura M. Tuschong, B.S., Erik M. Olson, B.S., Dennis D. Hickstein, M.D., David W. Russell, M.D., Ph.D.

Blood (2007) 110 (11): 3744.

https://doi.org/10.1182/blood.V110.11.3744.3744

Published: 2007

Journal Articles

Insertional Activation of GLI2 in Adult T-Cell Leukemia/Lymphoma.

Susan M. Cleveland, PhD, Utpal P. Dave, M.D.

Blood (2007) 110 (11): 4149.

https://doi.org/10.1182/blood.V110.11.4149.4149

Published: 2007

Journal Articles

Large Scale Copy Number Variation Upregulates the Expression of MYB in Human T-ALL.

Jennifer O’Neil, PhD, Joelle Tchinda, PhD, Alejandro Gutierrez, MD, Lisa Moreau, Keith McKenna, Richard Maser, PhD, Kwok-Kin Wong, MD, Wen-shu Wu, PhD, Ronald DePinho, MD, Lynda Chin, MD, Charles Lee, PhD, A. Thomas Look, MD

Blood (2006) 108 (11): 1408.

https://doi.org/10.1182/blood.V108.11.1408.1408

Published: 2006

Journal Articles

Biased Engraftment of Retrovirus-Transduced Marrow in Murine X-CGD Following Submyeloablative Conditioning.

Mohammed Sadat, Sara Dirscherl, Nancy Pech, Robert Getty, Scott Cross, Cecilia Barese, W. Scott Goebel, Mervin C. Yoder, Sean Mooney, Manuel Grez, Kenneth Cornetta, Mary C. Dinauer

Blood (2006) 108 (11): 3285.

https://doi.org/10.1182/blood.V108.11.3285.3285

Published: 2006

Journal Articles

Ex Vivo Expansion of Retrovirally-Transduced Primate CD34+ Cells Results in Preferential Engraftment and Persistence of Clones with MDS1/EVI1 Insertion Sites.

Theo Gomes, BS, Stephanie Sellers, BS, Robert E. Donahue, DVM, Rima Adler, BS., Andre La Rochelle, MD, PhD, Cynthia E. Dunbar, MD

Blood (2006) 108 (11): 203.

https://doi.org/10.1182/blood.V108.11.203.203

Published: 2006

Journal Articles

Identification of a Novel Core Promoter Element That Enhances Transcription: Application to Ankyrin Promoters for Globin Gene Therapy.

Karina Laflamme, PhD, Laura Elnitski, PhD, Ashley N. Owen, BA, Patrick G. Gallagher, MD, David M. Bodine, PhD

Blood (2006) 108 (11): 3261.

https://doi.org/10.1182/blood.V108.11.3261.3261

Published: 2006

Journal Articles

Insertional Activation of MDS1/EVI1, PRDM16 and SETBP1 in a Successful Chronic Granulomatous Disease (CGD) Gene Therapy Trial.

Kerstin Schwarzwaelder, Manfred Schmidt, PhD, Annette Deichmann, Marion G. Ott, MD, Stefan Stein, PhD, Hanno Glimm, MD, Ulrich Siler, MD, Dieter Hoelzer, MD, Reinhard Seger, MD, Manuel Grez, PhD, Christof von Kalle, MD

Blood (2006) 108 (11): 3274.

https://doi.org/10.1182/blood.V108.11.3274.3274

Published: 2006

Journal Articles

Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity

Ute Modlich, Jens Bohne, Manfred Schmidt, Christof von Kalle, Sabine Knöss, Axel Schambach, Christopher Baum

Blood (2006) 108 (8): 2545–2553.

https://doi.org/10.1182/blood-2005-08-024976

Published: 2006

Includes: Supplemental data

Journal Articles

Development of an In Vivo Exon Trapping Vector System To Detect Insertional Gene Activation by Lentiviral Vector.

Hideki Hanawa, MD, PhD, Takashi Shimada, MD, PhD

Blood (2005) 106 (11): 3059.

https://doi.org/10.1182/blood.V106.11.3059.3059

Published: 2005

NARROW

Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector

Overexpression of EVI1 Causes Genomic Instability and Cell Cycle Arrest in Hematopoietic Cells.

Lentiviral Vector Induced Insertional Haploinsufficiency of Ebf1 Causes Leukemia in a Murine Bone Marrow Transplantation Model

Deregulated EVI1 Expression Leads to Genomic Instability and G1 Cell Cycle Arrest

Integration-Mediated Activation of PRDM16 and HMGA2 in Multiple Clones without Adverse Hematopoietic Consequences Following Transplant of Autologous MGMTP140K Gene-Modified CD34 + Cells

Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

Notch Expression Expands the Pre-Malignant Pool of T-Cell Acute Lymphoblastic Leukemia Clones without Affecting Self-Renewal

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

Analysis of Viral Integration Sites in Human Induced Pluripotent Stem Cells.

Pre-Transplant Integration Site Diagnostics in Hematopoietic Stem Cell Gene Transfer.

Identification of a TFIID Binding Sequence That Increases Expression from the Ankyrin-1 Promoter: Application to Globin Gene Therapy

Reduced Genotoxic Risk Using Foamy Viral Vectors for the Treatment of Canine Leukocyte Adhesion Deficiency

Insertional Activation of GLI2 in Adult T-Cell Leukemia/Lymphoma.

Large Scale Copy Number Variation Upregulates the Expression of MYB in Human T-ALL.

Biased Engraftment of Retrovirus-Transduced Marrow in Murine X-CGD Following Submyeloablative Conditioning.

Ex Vivo Expansion of Retrovirally-Transduced Primate CD34+ Cells Results in Preferential Engraftment and Persistence of Clones with MDS1/EVI1 Insertion Sites.

Identification of a Novel Core Promoter Element That Enhances Transcription: Application to Ankyrin Promoters for Globin Gene Therapy.

Insertional Activation of MDS1/EVI1, PRDM16 and SETBP1 in a Successful Chronic Granulomatous Disease (CGD) Gene Therapy Trial.

Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity

Development of an In Vivo Exon Trapping Vector System To Detect Insertional Gene Activation by Lentiviral Vector.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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