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Journal Articles

TEM-MM-101: A Phase I/IIa Dose Escalation Study Evaluating the Safety and Activity of Autologous CD34+ Enriched Hematopoietic Progenitor Cells Genetically Modified for Human Interferon-α2 in Multiple Myeloma Patients with Early Relapse after Intensive Front Line Therapy

Bernhard Gentner, MD, Francesca Farina, MD, Matteo Giovanni Carrabba, MD, Magda Marcatti, MD, Stefania Girlanda, MD, Gabriele Antonarelli, MD, Capotondo Alessia, PhD, Stefania Mazzoleni, PhD, Carlo Russo, MD, Luigi Naldini, MD PhD, Fabio Ciceri, MD

Blood (2019) 134 (Supplement_1): 2064.

https://doi.org/10.1182/blood-2019-123749

Published: 2019

Journal Articles

Dissecting Ex Vivo Expansion of Mobilized Peripheral Blood Hematopoietic Stem and Progenitor Cells By Single Cell RNA Sequencing

Matteo Maria Naldini, Gabriele Casirati, MD, Erika Zonari, PhD, Giacomo Desantis, PhD, Andrea Cammarata, PhD, Stefano Beretta, PhD, Ivan Merelli, PhD, Fabio Ciceri, MD, Bernhard Gentner, MD

Blood (2018) 132 (Supplement 1): 3343.

https://doi.org/10.1182/blood-2018-99-111879

Published: 2018

Journal Articles

Thalassemia Gene Therapy By In Vivo Transduction of Mobilized Hematopoietic Stem Cells (HSCs) with an Integrating Hybrid Adenovirus Vector System

Afrodite Georgakopoulou, PhDstudent, Hongjie Wang, PhD, Chrysi Kapsali, Undergraduate, Nikoleta Psatha, PhD, Angeliki Koufali, Graduate, Chang Li, PhD, Zsuzsanna Izsvak, PhD, Achilles Anagnostopoulos, Thalia Papayannopoulou, MD DrSci, Evangelia Yannaki, MD, Andre Lieber, MD PhD

Blood (2018) 132 (Supplement 1): 2193.

https://doi.org/10.1182/blood-2018-99-116787

Published: 2018

Journal Articles

Umbilical Cord Blood Mononuclear Cells for Ex-Vivo Gene Therapy

Farid V. Bashirov, Ilnur I. Salafutdinov, PhD, Michail E. Sokolov, Andrew A. Izmailov, MD, Vage A. Markosyan, Filip O. Fadeev, Albert Rizvanov, DSc, PhD, Rustem I. Islamov

Blood (2018) 132 (Supplement 1): 5797.

https://doi.org/10.1182/blood-2018-99-113462

Published: 2018

Journal Articles

Preclinical Evaluation for the Gene Therapy of Patients with Leukocyte Adhesion Deficiency Type I

Elena Almarza, PhD, Cristina Mesa-Núñez, Carlos Damián, María Fernández-García, Begoña Díez-Cabezas, Jonathan D Schwartz, MD, Juan A Bueren

Blood (2018) 132 (Supplement 1): 5798.

https://doi.org/10.1182/blood-2018-99-111136

Published: 2018

Journal Articles

Enhanced Expression of CXCR4 Facilitates the Trafficking of Anti-c-Kit Chimeric Antigen Receptor (CAR) T Cells to Bone Marrow and Achieves Effective Donor Stem Cell Engraftment

Yasuyuki Arai, MDPhD, Cristina I. Corsino, Sherry M. Koontz, Masaki Tajima, Mary A. Black, Colin L. Sweeney, Uimook Choi, Steven A. Feldman, Mary C. Dinauer, Harry L Malech, MD

Blood (2017) 130 (Supplement 1): 4446.

https://doi.org/10.1182/blood.V130.Suppl_1.4446.4446

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

Ex Vivo Gene Therapy Approach By Targt Technology for the Treatment of β-Thalassemia Intermedia

Carla Casu, Vania Lo Presti, Emir M O'Hara, Garry Neil, Reem Miari, Nir Shapir, Stefano Rivella, PhD

Blood (2016) 128 (22): 2467.

https://doi.org/10.1182/blood.V128.22.2467.2467

Published: 2016

Journal Articles

Effective Therapy for Murine Hemophilia A by Enhancing Factor VIII Expression in Platelets

Xuefeng Wang, PhD, Fu Richard, Carol H. Miao, PhD

Blood (2015) 126 (23): 5547.

https://doi.org/10.1182/blood.V126.23.5547.5547

Published: 2015

Journal Articles

Bone Marrow Cell Based Enzyme Replacement Prolongs Survival and Improves Disease Phenotypes In a Mouse Model Of Lethal Hypophosphatasia

Osamu Iijima, PhD, Koichi Miyake, MD, PhD, Aki Nakamura, DDS, Tsutomu Igarashi, MD, PhD, Chizu Kanokoda, Atsushi Watanabe, MD, PhD, Takashi Shimada, MD, PhD

Blood (2013) 122 (21): 2897.

https://doi.org/10.1182/blood.V122.21.2897.2897

Published: 2013

Journal Articles

Murine Hemophilia A With Or Without Pre-Existing Anti-Factor VIII Inhibitors Is Partially Corrected By Factor VIII Stored In Platelets After Intraosseous Infusion Of Lentiviral Vectors Into Bone Marrow Without Preconditioning

Xuefeng Wang, PhD, Simon C. Shin, Chiang Andy, Dao Pan, PhD, David Rawlings, MD, Carol H. Miao, PhD

Blood (2013) 122 (21): 719.

https://doi.org/10.1182/blood.V122.21.719.719

Published: 2013

Journal Articles

Rescue of Lethal Hypophosphatasia Mice by Neonatal Ex Vivo Gene Therapy Using Lentivirally Transduced Bone Marrow Cells.

Osamu Iijima, PhD, Koichi Miyake, MD, PhD, Hanako Sugano-Tajima, MD, PhD, Tsutomu Igarashi, MD, PhD, Chizu Kanokoda, Atsushi Watanabe, MD, PhD, Takashi Shimada, MD, PhD

Blood (2012) 120 (21): 3154.

https://doi.org/10.1182/blood.V120.21.3154.3154

Published: 2012

Journal Articles

Induction of erythropoiesis using human vascular networks genetically engineered for controlled erythropoietin release

Ruei-Zeng Lin, Alexandra Dreyzin, Kristie Aamodt, Dan Li, Shou-Ching S. Jaminet, Andrew C. Dudley, Juan M. Melero-Martin

Blood (2011) 118 (20): 5420–5428.

https://doi.org/10.1182/blood-2011-08-372946

Published: 2011

Includes: Supplemental data

Journal Articles

Ex Vivo Gene Therapy for Haemophilia A: FVIII-Overexpressing Mesenchymal Stromal Cells Fail To Produce Sustained Therapeutic Levels of Circulating FVIII In Vivo .

Daniel L. Coutu, BSc, Moira Francois, Bsc, Jessica Cuerquis, Bsc, Hideto Matsui, MD,PhD, David Lillicrap, MD, Jacques Galipeau, MD

Blood (2006) 108 (11): 5472.

https://doi.org/10.1182/blood.V108.11.5472.5472

Published: 2006

Journal Articles

Ex Vivo Gene Trasfer for Hemophilia A That Enhances Safety and Results in Sustained In Vivo Factor VIII Expression from Lentivirally-Engineered Endothelial Progenitors.

Hideto Matsui, MD, PhD, Masaru Shibata, MD/PhD, Brian Brown, PhD, Andrea Labelle, Carol Hegadorn, Chandler Andrews, Robert P. Hebbel, MD, Jacques Galipeau, MD, Christine Hough, PhD, David Lillicrap, MD

Blood (2006) 108 (11): 3251.

https://doi.org/10.1182/blood.V108.11.3251.3251

Published: 2006

Journal Articles

Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)–deficient mice and corrects their immune and metabolic defects

Alessandra Mortellaro, Raisa Jofra Hernandez, Matteo M. Guerrini, Filippo Carlucci, Antonella Tabucchi, Maurilio Ponzoni, Francesca Sanvito, Claudio Doglioni, Clelia Di Serio, Luca Biasco, Antonia Follenzi, Luigi Naldini, Claudio Bordignon, Maria Grazia Roncarolo, Alessandro Aiuti

Blood (2006) 108 (9): 2979–2988.

https://doi.org/10.1182/blood-2006-05-023507

Published: 2006

Includes: Supplemental data

Journal Articles

Ex Vivo Gene Therapy of a Preadolescent with X-Linked Severe Combined Immunodeficiency.

Javier Chinen, MD, PhD, Jennifer M. Puck, MD, Joie Davis, RN, Gilda F. Linton, Med T, Narda L. Whiting-Theobald, BA, Patricia C. Woltz, RN, Rebecca H. Buckley, MD, Harry L. Malech, MD

Blood (2004) 104 (11): 410.

https://doi.org/10.1182/blood.V104.11.410.410

Published: 2004

Journal Articles

Integration of adeno-associated virus vectors in CD34+ human hematopoietic progenitor cells after transduction

G Fisher-Adams, KK Jr Wong, G Podsakoff, SJ Forman, S Chatterjee

Blood (1996) 88 (2): 492–504.

https://doi.org/10.1182/blood.V88.2.492.bloodjournal882492

Published: 1996

NARROW

TEM-MM-101: A Phase I/IIa Dose Escalation Study Evaluating the Safety and Activity of Autologous CD34+ Enriched Hematopoietic Progenitor Cells Genetically Modified for Human Interferon-α2 in Multiple Myeloma Patients with Early Relapse after Intensive Front Line Therapy

Dissecting Ex Vivo Expansion of Mobilized Peripheral Blood Hematopoietic Stem and Progenitor Cells By Single Cell RNA Sequencing

Thalassemia Gene Therapy By In Vivo Transduction of Mobilized Hematopoietic Stem Cells (HSCs) with an Integrating Hybrid Adenovirus Vector System

Umbilical Cord Blood Mononuclear Cells for Ex-Vivo Gene Therapy

Preclinical Evaluation for the Gene Therapy of Patients with Leukocyte Adhesion Deficiency Type I

Enhanced Expression of CXCR4 Facilitates the Trafficking of Anti-c-Kit Chimeric Antigen Receptor (CAR) T Cells to Bone Marrow and Achieves Effective Donor Stem Cell Engraftment

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

Ex Vivo Gene Therapy Approach By Targt Technology for the Treatment of β-Thalassemia Intermedia

Effective Therapy for Murine Hemophilia A by Enhancing Factor VIII Expression in Platelets

Bone Marrow Cell Based Enzyme Replacement Prolongs Survival and Improves Disease Phenotypes In a Mouse Model Of Lethal Hypophosphatasia

Murine Hemophilia A With Or Without Pre-Existing Anti-Factor VIII Inhibitors Is Partially Corrected By Factor VIII Stored In Platelets After Intraosseous Infusion Of Lentiviral Vectors Into Bone Marrow Without Preconditioning

Rescue of Lethal Hypophosphatasia Mice by Neonatal Ex Vivo Gene Therapy Using Lentivirally Transduced Bone Marrow Cells.

Induction of erythropoiesis using human vascular networks genetically engineered for controlled erythropoietin release

Ex Vivo Gene Therapy for Haemophilia A: FVIII-Overexpressing Mesenchymal Stromal Cells Fail To Produce Sustained Therapeutic Levels of Circulating FVIII In Vivo .

Ex Vivo Gene Trasfer for Hemophilia A That Enhances Safety and Results in Sustained In Vivo Factor VIII Expression from Lentivirally-Engineered Endothelial Progenitors.

Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)–deficient mice and corrects their immune and metabolic defects

Ex Vivo Gene Therapy of a Preadolescent with X-Linked Severe Combined Immunodeficiency.

Integration of adeno-associated virus vectors in CD34+ human hematopoietic progenitor cells after transduction

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American Society of Hematology

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American Society of Hematology

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