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Journal Articles

Foamy Virus Backbone Has Insulator Properties Which Remarkably Reduce Its Genotoxicity Potential

Michael Goodman, MD, Paritha Arumugam, PhD, Devin Pillis, MS, Danielle Lynn, MS, Johannes C.M. Van der Loo, PhD, Mehdi Keddache, PhD, Thomas R. Bauer, Jr., PhD, Dennis D. Hickstein, MD, David W Russell, MD PhD, Punam Malik, MD

Blood (2016) 128 (22): 1002.

https://doi.org/10.1182/blood.V128.22.1002.1002

Published: 2016

Journal Articles

RGS1 and RGS13 Regulate SDF-1-Mediated Responses and Homing in Human Cord Blood CD34+ Hematopoietic Stem/Progenitor Cells

Yorky Tsin Sik Wong, BSc, Kam Tong Leung, PhD, Karen Li, PhD, Kathy Yuen Yee Chan, PhD, Xiao-Bing Zhang, PhD, Tak Yeung Leung, MD, Kam Sze Tsang, PhD, Ting Fan Leung, MD, Pak Cheung Ng, MD FRCPCH, Chi Kong Li, MD

Blood (2015) 126 (23): 2371.

https://doi.org/10.1182/blood.V126.23.2371.2371

Published: 2015

Journal Articles

A Targeted Functional Clone Tracking Assay for the Identification of Tumour Suppressor Genes in BCP- ALL Implicates the Transcription Factors FOXO3 and PRDM1

Paul Sinclair, PhD, Joanna Cheng, Prahlad Raninga, Rebecca Hanna, Shaun Hollern, Amir Enshaei, PhD, Helen Blair, PhD, Sirintra Nakjang, Sarra Ryan, PhD, Jeyanthy Eswaran, PhD, Lars Buechler, Olaf Heidenreich, PhD, Christine Harrison, PhDFRCPath FMedSci

Blood (2015) 126 (23): 2449.

https://doi.org/10.1182/blood.V126.23.2449.2449

Published: 2015

Journal Articles

Optimization of Bcl11a Knockdown By miRNA Scaffold Embedded Shrnas Leading to Enhanced Induction of Fetal Hemoglobin in Erythroid Cells for the Treatment of Beta-Hemoglobinopathies

Christian Brendel, PhD, Swaroopa Guda, PhD, Raffaele Renella, MD PhD, Peng Du, PhD, Daniel E. Bauer, MD, PhD, Matthew C. Canver, BS, Sophia C. Kamran, MD, James Thornton, PhD, Helen de Boer, BAS, Michael D. Milsom, PhD, Stuart H. Orkin, MD, Richard Gregory, PhD, David A. Williams, MD

Blood (2014) 124 (21): 2150.

https://doi.org/10.1182/blood.V124.21.2150.2150

Published: 2014

Journal Articles

CDX2 regulates Human Leukemic Cell Clonogenicity and in Vivo Repopulation Capacity

Anna Paczulla, MSc, Sarah Grzywna, Matthias Grauer, Ursula Kohlhofer, Leticia Quintanilla-Martinez, MD, Lothar Kanz, MD, Claudia Lengerke, MD

Blood (2014) 124 (21): 1005.

https://doi.org/10.1182/blood.V124.21.1005.1005

Published: 2014

Journal Articles

The Effect of the R882H DNMT3a Mutation on Megakaryocyte Cell Lines

Yang Lu, MD, Dan Wang, Yifang Yuan, Li mei Chen, Yi ke Huang, Hao Huang, MD, Kefeng Shen, MD, Yanyan Ye, Jing Sun, MD, Qianli Jiang, MD PhD

Blood (2014) 124 (21): 5186.

https://doi.org/10.1182/blood.V124.21.5186.5186

Published: 2014

Journal Articles

Gene Therapy Corrects the Lethal Bone Marrow Failure in a Mouse Model for RPS19-Deficient Diamond-Blackfan Anemia

Pekka Jaako, Shubhranshu Debnath, Karin Olsson, Axel Schambach, MD, PhD, Christopher Baum, MD, Johan Flygare, MD, PhD, Stefan Karlsson, MD, PhD

Blood (2012) 120 (21): 513.

https://doi.org/10.1182/blood.V120.21.513.513

Published: 2012

Journal Articles

Hematopoietic SIN Lentiviral Micro RNA-Mediated Silencing of BCL11A: Pre-Clinical Evidence for a Sickle Cell Disease Gene-Therapy Trial

Raffaele Renella, MD, PhD, Aleksej Perlov, Chad E Harris, Daniel E. Bauer, MD, PhD, Jian Xu, PhD, Swaroopa Guda, PhD, Michael D. Milsom, PhD, Stuart H. Orkin, MD, David A. Williams, MD

Blood (2012) 120 (21): 753.

https://doi.org/10.1182/blood.V120.21.753.753

Published: 2012

Journal Articles

Lentiviral Transduction of Patient Derived Leukaemic Blasts Allowing In Vivo Bioluminescent Monitoring in An NSG Model of Leukaemia Stem Cell Maintenance,

Simon Bomken, MBBS, Lars Buechler, PhD, Klaus Rehe, MD, Frida Ponthan, PhD, Helen Blair, PhD, Olaf Heidenreich, PhD, Josef Vormoor, MD

Blood (2011) 118 (21): 4000.

https://doi.org/10.1182/blood.V118.21.4000.4000

Published: 2011

Journal Articles

Doxycycline Regulatable Expression of Cytidine Deaminase Mediates Myeloprotection and Avoids Lymphotoxicity in a Murine Transplant Model

Nico Lachmann, Sebastian Brennig, Nils Pfaff, Heiko Schermeier, Axel Schambach, MD PhD, Christopher Baum, MD, Thomas Moritz, MD

Blood (2011) 118 (21): 2054.

https://doi.org/10.1182/blood.V118.21.2054.2054

Published: 2011

Journal Articles

Gene Transfer to CD34+ Cells From Peripheral Blood Restores Osteoclast Function in Infantile Malignant Osteopetrosis

Ilana Moscatelli, PhD, Christian Thudium, Maria Askmyr, PhD, Ansgar S Schulz, MD, PhD, Oscar Porras, MD, PhD, Anders Fasth, MD, PhD, Kim Henriksen, PhD, Johan Richter, MD, PhD

Blood (2011) 118 (21): 2058.

https://doi.org/10.1182/blood.V118.21.2058.2058

Published: 2011

Journal Articles

Retroviral Ectopic Expression of a Signaling-Defective Thrombopoietin Receptor (Mpl) Induces a Systemic Loss of Hematopoietic Stem Cells in Mice,

Sabine Wintterle, MD, Dirk Heckl, PhD, Adrian Schwarzer, MD, Guntram Buesche, MD, Christopher Baum, MD, Ute Modlich, DVM, PhD

Blood (2011) 118 (21): 4175.

https://doi.org/10.1182/blood.V118.21.4175.4175

Published: 2011

Journal Articles

Transduction of Hematopoietic Stem/Progenitor Cells with a Lentivirus Expressing FANCA off the Human Phosphoglycerate Kinase Promoter Corrects the Fanconi Anemia Phenotype,

Zejin Sun, MD, PhD, Jingling Li, Yanzhu Yang, Yan Li, MD, PhD, Dirk Lindemann, Feng-chun Yang, MD, PhD, D. Wade Clapp, MD, Helmut Hanenberg, MD

Blood (2011) 118 (21): 4178.

https://doi.org/10.1182/blood.V118.21.4178.4178

Published: 2011

Journal Articles

The Ubiquitous Chromatin Opening Element (UCOE) Enhances Lentiviral Cytidine Deaminase (CDD) Expression and Drug Resistance During Hematopoietic Differentiation of Murine Induced Pluripotent Stem Cells (iPSCs),

Nils Pfaff, Nico Lachmann, Mania Ackermann, Saskia Kohlscheen, Christian Brendel, Michael Flasshove, MD, Axel Schambach, MD PhD, Thomas Moritz, MD

Blood (2011) 118 (21): 4179.

https://doi.org/10.1182/blood.V118.21.4179.4179

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

Impaired Lentiviral Transgene Expression In Vivo Caused by Massive Methylation of SFFV Promoter Sequences.

Friederike Herbst, Claudia R Ball, PhD, Francesca Tuorto, PhD, Wei Wang, Ulrich Kloz, Frank van der Hoeven, Frank Lyko, PhD, Christof Von Kalle, MD, Hanno Glimm, MD

Blood (2010) 116 (21): 3760.

https://doi.org/10.1182/blood.V116.21.3760.3760

Published: 2010

Journal Articles

Ectopic Expression of the Extracellular Domain of Mpl Is Sufficient to Induce a Hematopoietic Population Crisis

Daniel C. Wicke, Johann Meyer, Guntram Büsche, MD, Hans Kreipe, MD, Zhixiong Li, MD, Karl Welte, Matthias Ballmaier, Christopher Baum, Ute Modlich

Blood (2008) 112 (11): 2886.

https://doi.org/10.1182/blood.V112.11.2886.2886

Published: 2008

Journal Articles

High Level Production of a Lysosomal Enzyme from HSC-Derived Erythroid Cells for Therapeutic Correction of Hurler Syndrome in Mice.

Daren Wang, PhD, Wei Zhang, MS, Theodosia A. Kalfa, MD, PhD, Punam Malik, MD, Dao Pan, PhD

Blood (2008) 112 (11): 2355.

https://doi.org/10.1182/blood.V112.11.2355.2355

Published: 2008

Journal Articles

Treatment of Infections in Patients with X-Linked Chronic Granulomatous Disease (XCGD) with Gene Therapy.

Elizabeth M. Kang, MD, Gilda Linton, Narda Theobald, Sandra Anaya O’Brien, RN, Dianne Hilligoss, CNP, Harry L. Malech, MD

Blood (2007) 110 (11): 504.

https://doi.org/10.1182/blood.V110.11.504.504

Published: 2007

Journal Articles

High Efficiency Retro- and Lentiviral Transduction of Blood Endothelial Outgrowth Cells

Marcus A. Stockschlaeder, MD, Olga Shardakova, MD, Boris Fehse, PhD, Rudiger E. Scharf, MD

Blood (2007) 110 (11): 3727.

https://doi.org/10.1182/blood.V110.11.3727.3727

Published: 2007

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Foamy Virus Backbone Has Insulator Properties Which Remarkably Reduce Its Genotoxicity Potential

RGS1 and RGS13 Regulate SDF-1-Mediated Responses and Homing in Human Cord Blood CD34+ Hematopoietic Stem/Progenitor Cells

A Targeted Functional Clone Tracking Assay for the Identification of Tumour Suppressor Genes in BCP- ALL Implicates the Transcription Factors FOXO3 and PRDM1

Optimization of Bcl11a Knockdown By miRNA Scaffold Embedded Shrnas Leading to Enhanced Induction of Fetal Hemoglobin in Erythroid Cells for the Treatment of Beta-Hemoglobinopathies

CDX2 regulates Human Leukemic Cell Clonogenicity and in Vivo Repopulation Capacity

The Effect of the R882H DNMT3a Mutation on Megakaryocyte Cell Lines

Gene Therapy Corrects the Lethal Bone Marrow Failure in a Mouse Model for RPS19-Deficient Diamond-Blackfan Anemia

Hematopoietic SIN Lentiviral Micro RNA-Mediated Silencing of BCL11A: Pre-Clinical Evidence for a Sickle Cell Disease Gene-Therapy Trial

Lentiviral Transduction of Patient Derived Leukaemic Blasts Allowing In Vivo Bioluminescent Monitoring in An NSG Model of Leukaemia Stem Cell Maintenance,

Doxycycline Regulatable Expression of Cytidine Deaminase Mediates Myeloprotection and Avoids Lymphotoxicity in a Murine Transplant Model

Gene Transfer to CD34+ Cells From Peripheral Blood Restores Osteoclast Function in Infantile Malignant Osteopetrosis

Retroviral Ectopic Expression of a Signaling-Defective Thrombopoietin Receptor (Mpl) Induces a Systemic Loss of Hematopoietic Stem Cells in Mice,

Transduction of Hematopoietic Stem/Progenitor Cells with a Lentivirus Expressing FANCA off the Human Phosphoglycerate Kinase Promoter Corrects the Fanconi Anemia Phenotype,

The Ubiquitous Chromatin Opening Element (UCOE) Enhances Lentiviral Cytidine Deaminase (CDD) Expression and Drug Resistance During Hematopoietic Differentiation of Murine Induced Pluripotent Stem Cells (iPSCs),

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

Impaired Lentiviral Transgene Expression In Vivo Caused by Massive Methylation of SFFV Promoter Sequences.

Ectopic Expression of the Extracellular Domain of Mpl Is Sufficient to Induce a Hematopoietic Population Crisis

High Level Production of a Lysosomal Enzyme from HSC-Derived Erythroid Cells for Therapeutic Correction of Hurler Syndrome in Mice.

Treatment of Infections in Patients with X-Linked Chronic Granulomatous Disease (XCGD) with Gene Therapy.

High Efficiency Retro- and Lentiviral Transduction of Blood Endothelial Outgrowth Cells

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