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Journal Articles

Suicide Gene Therapy with a CD33 Targeted AAV6 Vector Expressing an Inducible Caspase-9 Suicide Gene Is Therapeutic in a Xenotransplantation Model of Acute Myeloid Leukemia

Nusrat Khan, MSc, Sridhar Bammidi, Jayandharan Giridhara Rao, PhD

Blood (2019) 134 (Supplement_1): 3351.

https://doi.org/10.1182/blood-2019-125222

Published: 2019

Journal Articles

T- and B-Cell Neogenesis Recovers Efficiently in Children with Acute Leukemia Given an Alpha-Beta T-Cell Depleted Haplo-HSCT Followed By Infusion of Donor T-Cells Genetically Modified with Inducible Caspase 9 Suicide Gene (BPX-501 cells)

Marialuigia Catanoso, MD, Emmanuel Clave, PhD, Corinne Douay, Mattia Algeri, MD, Isabelle Fournier, Valentina Bertaina, Itaua Leston Araujo, MSc, Francesca Del Bufalo, MD, Federica Galaverna, MD, Cecilia Surace, PhD, Alice Bertaina, MD PhD, Pietro Merli, MD, Aaron Foster, PhD, Antoine Toubert, MD PhD, Franco Locatelli

Blood (2018) 132 (Supplement 1): 4560.

https://doi.org/10.1182/blood-2018-99-116536

Published: 2018

Journal Articles

Phase I-IIa Clinical Trial to Assess Safety and Efficacy of MLM-CAR44.1, a CD44v6 Directed CAR-T in Relapsed/Refractory Acute Myeloid Leukemia (AML) and Multiple Myeloma (MM)

Matteo Giovanni Carrabba, MD, Monica Casucci, PhD, Michael Hudecek, MD, Concetta Quintarelli, PhD, Javier Briones, MD, Roman Hajek, MD PhD, Jorge Sierra, MD, Franco Locatelli, Hermann Einsele, MD PhD, Claudio Bordignon, MD, Fabio Ciceri, MD, Catia Traversari, PhD

Blood (2018) 132 (Supplement 1): 5790.

https://doi.org/10.1182/blood-2018-99-117974

Published: 2018

Journal Articles

A Novel Pharmacologic 'Remote Control' to Steer CAR-T Cell Function In Vivo

Katrin Mestermann, Julian Rydzek, Frenz Silke, Thomas Nerreter, Mades Andreas, Hermann Einsele, MD, Michael Hudecek, MD

Blood (2018) 132 (Supplement 1): 2055.

https://doi.org/10.1182/blood-2018-99-118626

Published: 2018

Journal Articles

Donor T Cells Genetically Modified with a Novel Suicide Gene (inducible Caspase 9, iC9) Expand and Persist over Time after Post-Allograft Infusion in Patients Given αβ T-Cell and B-Cell Depleted HLA-Haploidentical Allogeneic Stem Cell Transplantation (αβ haplo-HSCT) Contributing to Accelerate Immune Recovery

Pietro Merli, MD, Valentina Bertaina, Federica Galaverna, MD, Mattia Algeri, MD, Matilde Sinibaldi, Luisa Strocchio, Giuseppina Li Pira, Daria Pagliara, Sonia De Murtas, Giuseppe Palumbo, Vincent O'Neill, David M. Spencer, Aaron Foster, Alice Bertaina, MD PhD, Franco Locatelli, MD PhD

Blood (2017) 130 (Supplement 1): 211.

https://doi.org/10.1182/blood.V130.Suppl_1.211.211

Published: 2017

Journal Articles

Anti-CD19 Chimeric Antigen Receptor in Hematopoietic Stem Cells Controlled By the Suicide Gene HSV-sr39TK for Immunotherapy of B-Lineage Malignancies

Laurel Christine Truscott, MD, Tzu Ting Chiou, PhD, Satiro N. De Oliveira, MD

Blood (2016) 128 (22): 2317.

https://doi.org/10.1182/blood.V128.22.2317.2317

Published: 2016

Journal Articles

Clinical Outcome and Immune Recovery after Adoptive Infusion of BPX-501 Cells (donor T cells transduced with iC9 suicide gene) in Children with Hemoglobinopathies and Diamond-Blackfan Anemia Given α/β T-Cell Depleted HLA-Haploidentical Stem Cell Transplantation (HSCT)

Alice Bertaina, MDPhD, Pietro Merli, MD, Daria Pagliara, MD, Giuseppina Li Pira, PhD, Biagio De Angelis, PhD, Letizia Pomponia Brescia, MD, Luisa Strocchio, MD, Francesca Del Bufalo, MD, Mauro Montanari, MD, Barbarella Lucarelli, MD, Mattia Algeri, MD, Martha French, PhD, Annemarie Moseley, MDPhD, Franco Locatelli, MD

Blood (2016) 128 (22): 2286.

https://doi.org/10.1182/blood.V128.22.2286.2286

Published: 2016

Journal Articles

Outcome of Children with Primary Immune-Deficiencies (PIDs) Enrolled in a Phase I-II Trial Based on the Infusion of BPX-501 Donor T Cells Genetically Modified with a Novel Suicide Gene (inducible Caspase 9, iC9) after T-Cell Depleted HLA-Haploidentical Allogeneic Stem Cell Transplantation (haplo-HSCT)

Neena Kapoor, MD, Alice Bertaina, MD PhD, Pietro Merli, MD, Swati Naik, MD, Waseem Qasim, MBBS PhD, Mary Slatter, MD, Reem Elfeky, MD, Barbarella Lucarelli, MD, Monika Smogorzewska, PhD, Giuseppina Li Pira, PhD, Valentina Bertaina, PhD, Annemarie Moseley, MD PhD, Franco Locatelli, MD

Blood (2016) 128 (22): 72.

https://doi.org/10.1182/blood.V128.22.72.72

Published: 2016

Journal Articles

T-Cell Depleted HLA-Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (haplo-HSCT) Followed By Donor Lymphocyte Infusion with T Cells Transduced with the Inducible Caspase 9 (iC9) Suicide Gene in Children with Hematological Malignancies

Barbarella Lucarelli, MD, Alice Bertaina, MD PhD, Pietro Merli, MD, Concetta Quintarelli, PhD, Daniela Pende, MD, Michela Falco, PhD, Maria Giuseppina Cefalo, MD, Giuseppina Li Pira, PhD, Ignazio Caruana, PhD, Letizia Pomponia Brescia, MD, Luisa Strocchio, MD, Giuseppe Maria Milano, MD, Valentina Bertaina, PhD, Matilde Sinibaldi, PhD, Mattia Algeri, MD, Lorenzo Moretta, MD, Joanna Stanson, Annemarie Moseley, MD PhD, Franco Locatelli, MD

Blood (2016) 128 (22): 4683.

https://doi.org/10.1182/blood.V128.22.4683.4683

Published: 2016

Journal Articles

Impact of Immune Reconstitution (IR) and Graft-Versus-Host Disease (GvHD) on Clinical Outcomes after Treatment with Donor T Cells Transduced to Express the Herpes Simplex Virus Thymidine-Kinase Suicide Gene (TK cells) in Acute Leukemia Patients Undergoing Haploidentical Hematopoietic Stem Cell Transplantation (HSCT)

Blood (2016) 128 (22): 4599.

https://doi.org/10.1182/blood.V128.22.4599.4599

Published: 2016

Journal Articles

Efficient Site-Specific Multi-Gene Engineering of Renewable Pluripotent Cells for Generation of Off-the-Shelf Hematopoietic Immunotherapeutics

Ramzey Abujarour, PhD, Weijie Lan, PhD, Tom Tong Lee, PhD, Greg Bonello, Miguel Meza, MS, Megan Robinson, BS, Raedun Clarke, PhD, Christopher Truong, Chris Lynn, BS, David Robbins, BS, Betsy Rezner, BS, Stewart E. Abbot, PhD BSc, MSc, Daniel Shoemaker, PhD, Bahram Valamehr, PhD MBA

Blood (2016) 128 (22): 5887.

https://doi.org/10.1182/blood.V128.22.5887.5887

Published: 2016

Journal Articles

Gene Modification of Human Hematopoietic Stem Cells with Suicide Genes As a Safety Control

Laurel Christine Truscott, MD, Tzu Ting Chiou, PhD, Roy L. Kao, MD, Satiro N. De Oliveira, MD

Blood (2016) 128 (22): 5888.

https://doi.org/10.1182/blood.V128.22.5888.5888

Published: 2016

Journal Articles

Tracking Genetically Engineered Lymphocytes Long-Term Reveals the Dynamics of T-Cell Immunological Memory

Blood (2015) 126 (23): 263.

https://doi.org/10.1182/blood.V126.23.263.263

Published: 2015

Journal Articles

BPX-501 Cells (donor T cells transduced with iC9 suicide gene) Are Able to Clear Life-Threatening Viral Infections in Children with Primary Immune Deficiencies Given Alpha/Beta T-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (haplo-HSCT)

Barbarella Lucarelli, MD, Pietro Merli, MD, Alice Bertaina, MDPhD, Matilde Sinibaldi, PhD, Stefania Gaspari, MD, Luisa Strocchio, MD, Angela Pitisci, PhD, Giuseppina Li Pira, PhD, Giuseppe Maria Milano, MD, Annemarie Moseley, MDPhD, Concetta Quintarelli, PhD, Franco Locatelli, Prof, MD PhD

Blood (2015) 126 (23): 4299.

https://doi.org/10.1182/blood.V126.23.4299.4299

Published: 2015

Journal Articles

Human Induced Pluripotent Stem Cells Incorporating Safe Harbor Loci Integrated Inducible Suicide Systems for Use in the Application of Cellular Therapeutics

Xiaosong Huang, PhD, Weijie Lan, PhD, Philippe Parone, PhD, Raedun Clarke, PhD, Ramzey Abujarour, PhD, Megan Robinson, BS, Miguel Meza, MS, Sarah Raynel, MS, Betsy Rezner, BS, David Robbins, BS, Tom Tong Lee, PhD, Daniel Shoemaker, PhD, Bahram Valamehr, PhD MBA

Blood (2015) 126 (23): 3237.

https://doi.org/10.1182/blood.V126.23.3237.3237

Published: 2015

Journal Articles

Clinical Outcome after Adoptive Infusion of BPX-501 Cells (donor T cells transduced with iC9 suicide gene) in Children Given Alpha/Beta T-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (haplo-HSCT): Preliminary Results of a Phase I-II Trial

Franco Locatelli, Prof, MD PhD, Pietro Merli, MD, Giuseppina Li Pira, PhD, Valentina Bertaina, PhD, Barbarella Lucarelli, MD, Letizia Pomponia Brescia, MD, Mauro Montanari, MD, Daniela Pende, MD, Michela Falco, PhD, Daria Pagliara, MD, Maria Giuseppina Cefalo, MD, Alessandro Moretta, MD, Concetta Quintarelli, PhD, Brigitte Strahm, MD, Waseem Qasim, MBBS PhD, Mary Slatter, Lorenzo Moretta, MD, Annemarie Moseley, MDPhD, Alice Bertaina, MDPhD

Blood (2015) 126 (23): 1931.

https://doi.org/10.1182/blood.V126.23.1931.1931

Published: 2015

Journal Articles

Immune Reconstitution after Adoptive Infusion of BPX501 Cells (donor T cells transduced with iC9 suicide gene) in Children Given Alpha/Beta T-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (haplo-HSCT): Preliminary Phenotypic and Functional Results of a Phase I-II Trial

Pietro Merli, MD, Giuseppina Li Pira, PhD, Valentina Bertaina, PhD, Matilde Sinibaldi, PhD, Barbarella Lucarelli, MD, Letizia Pomponia Brescia, MD, Giuseppe Palumbo, MD, Giuseppe Maria Milano, MD, Maria Teresa Romano, MD, Valentina Cirillo, PhD, Luisa Strocchio, MD, Irma Airoldi, PhD, Luciana Vinti, MD, Concetta Quintarelli, PhD, Lorenzo Moretta, MD, Annemarie Moseley, MDPhD, Alice Bertaina, MDPhD, Franco Locatelli, Prof, MD PhD

Blood (2015) 126 (23): 3093.

https://doi.org/10.1182/blood.V126.23.3093.3093

Published: 2015

Journal Articles

Establishing Modified Human CYP4B1 As a Novel Suicide Gene System for Adoptive T-Cell Therapies

Katharina Roellecke, M.Sc., Constanze Wiek, PhD, Christof Kramm, MD, Allan Rettie, PhD, Helmut Hanenberg, MD

Blood (2015) 126 (23): 5550.

https://doi.org/10.1182/blood.V126.23.5550.5550

Published: 2015

Journal Articles

Inducible caspase-9 suicide gene controls adverse effects from alloreplete T cells after haploidentical stem cell transplantation

Xiaoou Zhou, Gianpietro Dotti, Robert A. Krance, Caridad A. Martinez, Swati Naik, Rammurti T. Kamble, April G. Durett, Olga Dakhova, Barbara Savoldo, Antonio Di Stasi, David M. Spencer, Yu-Feng Lin, Hao Liu, Bambi J. Grilley, Adrian P. Gee, Cliona M. Rooney, Helen E. Heslop, Malcolm K. Brenner

Blood (2015) 125 (26): 4103–4113.

https://doi.org/10.1182/blood-2015-02-628354

Published: 2015

Includes: Supplemental data

Journal Articles

Infusion of Donor Lymphocytes Genetically Engineered to Express the Herpes Simplex Virus Thymidine Kinase (HSV-TK) Suicide Gene after Haploidentical Hematopoietic Stem Cell Transplantation (HSCT): Preliminary Efficacy Data from the Randomized TK008 Study

Blood (2014) 124 (21): 2535.

https://doi.org/10.1182/blood.V124.21.2535.2535

Published: 2014

NARROW

Suicide Gene Therapy with a CD33 Targeted AAV6 Vector Expressing an Inducible Caspase-9 Suicide Gene Is Therapeutic in a Xenotransplantation Model of Acute Myeloid Leukemia

T- and B-Cell Neogenesis Recovers Efficiently in Children with Acute Leukemia Given an Alpha-Beta T-Cell Depleted Haplo-HSCT Followed By Infusion of Donor T-Cells Genetically Modified with Inducible Caspase 9 Suicide Gene (BPX-501 cells)

Phase I-IIa Clinical Trial to Assess Safety and Efficacy of MLM-CAR44.1, a CD44v6 Directed CAR-T in Relapsed/Refractory Acute Myeloid Leukemia (AML) and Multiple Myeloma (MM)

A Novel Pharmacologic 'Remote Control' to Steer CAR-T Cell Function In Vivo

Anti-CD19 Chimeric Antigen Receptor in Hematopoietic Stem Cells Controlled By the Suicide Gene HSV-sr39TK for Immunotherapy of B-Lineage Malignancies

Clinical Outcome and Immune Recovery after Adoptive Infusion of BPX-501 Cells (donor T cells transduced with iC9 suicide gene) in Children with Hemoglobinopathies and Diamond-Blackfan Anemia Given α/β T-Cell Depleted HLA-Haploidentical Stem Cell Transplantation (HSCT)

T-Cell Depleted HLA-Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (haplo-HSCT) Followed By Donor Lymphocyte Infusion with T Cells Transduced with the Inducible Caspase 9 (iC9) Suicide Gene in Children with Hematological Malignancies

Efficient Site-Specific Multi-Gene Engineering of Renewable Pluripotent Cells for Generation of Off-the-Shelf Hematopoietic Immunotherapeutics

Gene Modification of Human Hematopoietic Stem Cells with Suicide Genes As a Safety Control

Tracking Genetically Engineered Lymphocytes Long-Term Reveals the Dynamics of T-Cell Immunological Memory

BPX-501 Cells (donor T cells transduced with iC9 suicide gene) Are Able to Clear Life-Threatening Viral Infections in Children with Primary Immune Deficiencies Given Alpha/Beta T-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (haplo-HSCT)

Human Induced Pluripotent Stem Cells Incorporating Safe Harbor Loci Integrated Inducible Suicide Systems for Use in the Application of Cellular Therapeutics

Clinical Outcome after Adoptive Infusion of BPX-501 Cells (donor T cells transduced with iC9 suicide gene) in Children Given Alpha/Beta T-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (haplo-HSCT): Preliminary Results of a Phase I-II Trial

Immune Reconstitution after Adoptive Infusion of BPX501 Cells (donor T cells transduced with iC9 suicide gene) in Children Given Alpha/Beta T-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (haplo-HSCT): Preliminary Phenotypic and Functional Results of a Phase I-II Trial

Establishing Modified Human CYP4B1 As a Novel Suicide Gene System for Adoptive T-Cell Therapies

Inducible caspase-9 suicide gene controls adverse effects from alloreplete T cells after haploidentical stem cell transplantation

Infusion of Donor Lymphocytes Genetically Engineered to Express the Herpes Simplex Virus Thymidine Kinase (HSV-TK) Suicide Gene after Haploidentical Hematopoietic Stem Cell Transplantation (HSCT): Preliminary Efficacy Data from the Randomized TK008 Study

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American Society of Hematology

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American Society of Hematology

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