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Journal Articles

Novel Lentiviral Vectors for Gene Therapy of Sickle Cell Disease Combining Gene Addition and Gene Silencing Strategies

Megane Brusson, Anne Chalumeau, Pierre Martinucci, Valentina Poletti, Fulvio Mavilio, Marina Cavazzana, Mario Amendola, Annarita Miccio

Blood (2021) 138 (Supplement 1): 3973.

https://doi.org/10.1182/blood-2021-151076

Published: 2021

Journal Articles

Clinical Results of the Drepaglobe Trial for Sickle Cell Disease Patients

Blood (2021) 138 (Supplement 1): 1854.

https://doi.org/10.1182/blood-2021-152331

Published: 2021

Journal Articles

Editing a γ-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Phenotype of Sickle Cell Disease Erythrocytes

Leslie Weber, Giacomo Frati, Tristan Felix, Clara Wollenschlaeger, Antonio Casini, Vasco Meneghini, Cecile Masson, Anne De Cian, Anne Chalumeau, Fulvio Mavilio, PhD, Mario Amendola, Isabelle André, PhD, Anna Cereseto, Wassim El Nemer, PhD, Jean-Paul Concordet, Carine Giovannangeli, Marina Cavazzana, Annarita Miccio, PhD

Blood (2019) 134 (Supplement_1): 4635.

https://doi.org/10.1182/blood-2019-123192

Published: 2019

Journal Articles

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

Chiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, Tristan Felix, Oriana Romano, Elisa Magrin, Leslie Weber, Giulia Pavani, Sara El Hoss, Ryo Kurita, Yukio Nakamura, Thomas J. Cradick, Ante S. Lundberg, Matthew Porteus, Mario Amendola, Wassim El Nemer, Marina Cavazzana, Fulvio Mavilio, Annarita Miccio

Blood (2018) 131 (17): 1960–1973.

https://doi.org/10.1182/blood-2017-10-811505

Published: 2018

Includes: Supplemental data

Journal Articles

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

Blood (2017) 130 (11): 1327–1335.

https://doi.org/10.1182/blood-2017-04-777136

Published: 2017

Includes: Supplemental data

Journal Articles

Induction of Fetal Hemoglobin Synthesis By Crispr/Cas9-Mediated Disruption of the β-Globin Locus Architecture

Chiara Antoniani, MSc, Vasco Meneghini, Annalisa Lattanzi, PhD, Giulia Pavani, PhD, Tristan Felix, MSc, Fatima Amor, MSc, Oriana Romano, Elisa Magrin, PhD, Leslie Weber, MSc, Thomas Cradick, Ante S Lundberg, MD, Matthew Porteus, MD PhD, Mario Amendola, Marina Cavazzana, MD PhD, Fulvio Mavilio, PhD, Annarita Miccio, PhD

Blood (2016) 128 (22): 321.

https://doi.org/10.1182/blood.V128.22.321.321

Published: 2016

Journal Articles

Preclinical Development of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1)

Valentina Poletti, PhD, Sabine Charrier, PhD, Samia Martin, Bernard Gjata, Alban Vignaud, PhD, Fang Zhang, PhD, Karen Buckland, PhD, Michael Rothe, PhD, Axel Schambach, MD PhD, Sung-Yun Pai, MD, David A. Williams, MD, H. Bobby Gaspar, MD PhD, Adrian J Thrasher, MD PhD, Fulvio Mavilio, PhD

Blood (2016) 128 (22): 4705.

https://doi.org/10.1182/blood.V128.22.4705.4705

Published: 2016

Journal Articles

Re-Creating Hereditary Persistence of Fetal Hemoglobin (HPFH) to Treat Sickle Cell Disease (SCD) and β-Thalassemia

Blood (2016) 128 (22): 4708.

https://doi.org/10.1182/blood.V128.22.4708.4708

Published: 2016

Journal Articles

Gene Editing with Crispr-Cas9 for Treating Beta-Hemoglobinopathies

Ciaran Lee, PhD, Gang Bao, PhD, Matthew H. Porteus, MD PhD, Tatjana Cornu, PhD, Annarita Miccio, PhD, Thomas Cradick, Toni Cathomen, PhD, Ante Lundberg, MD, Fulvio Mavilio, PhD

Blood (2015) 126 (23): 3376.

https://doi.org/10.1182/blood.V126.23.3376.3376

Published: 2015

Journal Articles

IL-7 and IL-15 instruct the generation of human memory stem T cells from naive precursors

Nicoletta Cieri, Barbara Camisa, Fabienne Cocchiarella, Mattia Forcato, Giacomo Oliveira, Elena Provasi, Attilio Bondanza, Claudio Bordignon, Jacopo Peccatori, Fabio Ciceri, Maria Teresa Lupo-Stanghellini, Fulvio Mavilio, Anna Mondino, Silvio Bicciato, Alessandra Recchia, Chiara Bonini

Blood (2013) 121 (4): 573–584.

https://doi.org/10.1182/blood-2012-05-431718

Published: 2013

Includes: Supplemental data

Journal Articles

Abnormalities of the Hematopoietic Stem Cell Compartment in Children After in Utero Exposure to AZT

Isabelle Andre-Schmutz, PhD, Liliane Dal Cortivo, Emmanuelle M. Six, PhD, Sophie Kaltenbach, Isabelle Radford-Weiss, Nicolas Cagnard, Fabienne Cocchiarella, Alexandra Benachi, Laurent Mandelbrot, Fulvio Mavilio, Marina Cavazzana-Calvo, MD, PhD, Stéphane Blanche, MD

Blood (2011) 118 (21): 1123.

https://doi.org/10.1182/blood.V118.21.1123.1123

Published: 2011

Journal Articles

The Importance of Be(ginn)Ing Naïve: Implications for Cancer Immune-Gene Therapy

Nicoletta Cieri, MD, Barbara Camisa, Fabienne Cocchiarella, Elena Provasi, PhD, Zulma Magnani, Attilio Bondanza, MD, PhD, Anna Mondino, PhD, Fulvio Mavilio, PhD, Silvio Bicciato, PhD, Alessandra Recchia, PhD, Chiara Bonini

Blood (2011) 118 (21): 2052.

https://doi.org/10.1182/blood.V118.21.2052.2052

Published: 2011

Journal Articles

High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors

Claudia Cattoglio, Danilo Pellin, Ermanno Rizzi, Giulietta Maruggi, Giorgio Corti, Francesca Miselli, Daniela Sartori, Alessandro Guffanti, Clelia Di Serio, Alessandro Ambrosi, Gianluca De Bellis, Fulvio Mavilio

Blood (2010) 116 (25): 5507–5517.

https://doi.org/10.1182/blood-2010-05-283523

Published: 2010

Includes: Supplemental data

Journal Articles

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy

Barbara Cassani, Eugenio Montini, Giulietta Maruggi, Alessandro Ambrosi, Massimiliano Mirolo, Silvia Selleri, Erika Biral, Ilaria Frugnoli, Vivian Hernandez-Trujillo, Clelia Di Serio, Maria Grazia Roncarolo, Luigi Naldini, Fulvio Mavilio, Alessandro Aiuti

Blood (2009) 114 (17): 3546–3556.

https://doi.org/10.1182/blood-2009-02-202085

Published: 2009

Includes: Supplemental data

Journal Articles

Transcription Factor Binding Sites Are Genetic Determinant of Retroviral Integration in the Human Genome

Claudia Cattoglio, Barbara Felice, Davide Cittaro, Annarita Miccio, Giuliana Ferrari, Alessandra Recchia, Lucilla Luzi, Fulvio Mavilio

Blood (2008) 112 (11): 820.

https://doi.org/10.1182/blood.V112.11.820.820

Published: 2008

Journal Articles

Comprehensive and Unbiased Integration Site Analysis in Clinical Gene Therapy.

Blood (2008) 112 (11): 2351.

https://doi.org/10.1182/blood.V112.11.2351.2351

Published: 2008

Journal Articles

Gamma-Retroviral and Lentiviral Vectors Have Different Preferences for Specific Classes of Genes and Specific Genomic Features in Human Hematopoietic Cells

Alessandra Recchia, PhD, Anna Testa, PhD, Davide Cittaro, Barbara Felice, Claudia Cattoglio, Lucilla Luzi, PhD, Fulvio Mavilio, PhD

Blood (2007) 110 (11): 3732.

https://doi.org/10.1182/blood.V110.11.3732.3732

Published: 2007

Journal Articles

Hot spots of retroviral integration in human CD34 + hematopoietic cells

Claudia Cattoglio, Giulia Facchini, Daniela Sartori, Antonella Antonelli, Annarita Miccio, Barbara Cassani, Manfred Schmidt, Christof von Kalle, Steve Howe, Adrian J. Thrasher, Alessandro Aiuti, Giuliana Ferrari, Alessandra Recchia, Fulvio Mavilio

Blood (2007) 110 (6): 1770–1778.

https://doi.org/10.1182/blood-2007-01-068759

Published: 2007

Includes: Supplemental data

Journal Articles

Clonal Analyses of Retroviral Vector Integrations in ADA-SCID Patients Treated with Stem Cell Gene Therapy.

Barbara Cassani, Grazia Andolfi, Massimiliano Mirolo, Luca Biasco, Alessandra Recchia, Fabrizia Urbinati, Cristina Valacca, Samantha Scaramuzza, Matteo Cazzola, Daniela Sartori, Alessandro Ambrosi, Clelia Di Serio, Maria Grazia Roncarolo, Fulvio Mavilio, Claudio Bordignon, Alessandro Aiuti

Blood (2006) 108 (11): 3249.

https://doi.org/10.1182/blood.V108.11.3249.3249

Published: 2006

Journal Articles

Retroviral Vector Integration Deregulates Gene Expression but Has No Consequences on the Biology and Function of Transplanted T Cells.

Alessandra Recchia, PhD, Chiara Bonini, MD, Zulma Magnani, Fabrizia Urbinati, PhD, Daniela Sartori, Sara Muraro, Fabio Ciceri, MD, Claudio Bordignon, MD, Fulvio Mavilio, PhD

Blood (2005) 106 (11): 462.

https://doi.org/10.1182/blood.V106.11.462.462

Published: 2005

NARROW

Novel Lentiviral Vectors for Gene Therapy of Sickle Cell Disease Combining Gene Addition and Gene Silencing Strategies

Clinical Results of the Drepaglobe Trial for Sickle Cell Disease Patients

Editing a γ-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Phenotype of Sickle Cell Disease Erythrocytes

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

Induction of Fetal Hemoglobin Synthesis By Crispr/Cas9-Mediated Disruption of the β-Globin Locus Architecture

Preclinical Development of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1)

Re-Creating Hereditary Persistence of Fetal Hemoglobin (HPFH) to Treat Sickle Cell Disease (SCD) and β-Thalassemia

Gene Editing with Crispr-Cas9 for Treating Beta-Hemoglobinopathies

IL-7 and IL-15 instruct the generation of human memory stem T cells from naive precursors

Abnormalities of the Hematopoietic Stem Cell Compartment in Children After in Utero Exposure to AZT

The Importance of Be(ginn)Ing Naïve: Implications for Cancer Immune-Gene Therapy

High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy

Transcription Factor Binding Sites Are Genetic Determinant of Retroviral Integration in the Human Genome

Comprehensive and Unbiased Integration Site Analysis in Clinical Gene Therapy.

Gamma-Retroviral and Lentiviral Vectors Have Different Preferences for Specific Classes of Genes and Specific Genomic Features in Human Hematopoietic Cells

Hot spots of retroviral integration in human CD34 + hematopoietic cells

Clonal Analyses of Retroviral Vector Integrations in ADA-SCID Patients Treated with Stem Cell Gene Therapy.

Retroviral Vector Integration Deregulates Gene Expression but Has No Consequences on the Biology and Function of Transplanted T Cells.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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