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Journal Articles

Enrichment of Hematopoietic Stem Cells Using Immunomagnetic Beads to Facilitate Gene Therapy

Katelyn E Masiuk, BS, Devin Brown, BA, Fabrizia Urbinati, PhD, Roger P Hollis, PhD, Donald B Kohn

Blood (2016) 128 (22): 2313.

https://doi.org/10.1182/blood.V128.22.2313.2313

Published: 2016

Journal Articles

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

Blood (2015) 125 (17): 2597–2604.

https://doi.org/10.1182/blood-2014-12-615948

Published: 2015

Includes: Supplemental data

Journal Articles

Zinc Finger Nucleases Targeting The β-Globin Locus Drive Efficient Correction Of The Sickle Mutation In CD34 + Cells

Megan D Hoban, BS, Alok V Joglekar, PhD, David Gray, BS, Michael L Kaufman, MS, Fabrizia Urbinati, PhD, Shantha Senadheera, MS, Gregory Cost, PhD, Andreas Reik, PhD, Michael C. Holmes, PhD, Philip D Gregory, PhD, Roger P. Hollis, PhD, Donald B. Kohn, MD

Blood (2013) 122 (21): 2904.

https://doi.org/10.1182/blood.V122.21.2904.2904

Published: 2013

Journal Articles

Increasing Transduction Efficiency in Human Hematopoietic Stem Cells for Gene Therapy

Kismet M Baldwin, MD, Fabrizia Urbinati, Ph.D, Zulema Romero-Garcia, Ph D, Donald B. Kohn, MD

Blood (2012) 120 (21): 2052.

https://doi.org/10.1182/blood.V120.21.2052.2052

Published: 2012

Journal Articles

Laser Tweezers Raman Spectroscopy As a Novel Red Blood Cell Functional Assay for Sickle Cell Disease

Rui Liu, Lena Zheng, Denis M. Dwyre, MD, Fabrizia Urbinati, Ph.D, Donald B. Kohn, MD, Dennis Matthews, James Chan, PhD, Noriko Satake, MD

Blood (2011) 118 (21): 4847.

https://doi.org/10.1182/blood.V118.21.4847.4847

Published: 2011

Journal Articles

Preclinical Studies for Sickle Cell Disease Gene Therapy Using Bone Marrow CD34+ Cells Modified with a βAS3-Globin Lentiviral Vector

Fabrizia Urbinati, Ph.D, Zulema Romero Garcia, Ph.D, Sabine Geiger, Rafael Ruiz de Assin, Ph.D, Gabriela Kuftinec, Roger P. Hollis, PhD, Satiro N. De Oliveira, MD, Jennifer Wherley, Aaron Cooper, Tim M. Townes, PhD, Gay M. Crooks, MBBS, Elizabeth J Read, MD, Mark C. Walters, MD, Herbert J. Meiselman, ScD, Thomas D. Coates, MD, Victor J. Marder, M.D., Donald B. Kohn, MD

Blood (2011) 118 (21): 3119.

https://doi.org/10.1182/blood.V118.21.3119.3119

Published: 2011

Journal Articles

A Novel Combination of Chicken Hypersensitive Site-4 Insulator Elements Improves Titers and Restores Full Insulator Activity.

Paritha Arumugam, Fabrizia Urbinati, Phd., Chinavenmeni S. Velu, PhD, Tomoyasu Higashimoto, Phd., H. Leighton Grimes, PhD, Punam Malik

Blood (2009) 114 (22): 3566.

https://doi.org/10.1182/blood.V114.22.3566.3566

Published: 2009

Journal Articles

A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction

Ajay Perumbeti, Tomoyasu Higashimoto, Fabrizia Urbinati, Robert Franco, Herbert J. Meiselman, David Witte, Punam Malik

Blood (2009) 114 (6): 1174–1185.

https://doi.org/10.1182/blood-2009-01-201863

Published: 2009

Includes: Supplemental data

Journal Articles

The 3′ End of the Chicken Hypersensitive Site-4 Insulator Has Properties Similar to the 5′ Insulator Core and Is Necessary in Conjunction with the Core for Full Insulator Activity

Paritha Arumugam, Fabrizia Urbinati, PhD., Chinavenmeni S. Velu, PhD, H. Leighton Grimes, PhD, Punam Malik, MD

Blood (2008) 112 (11): 817.

https://doi.org/10.1182/blood.V112.11.817.817

Published: 2008

Journal Articles

Mechanism of Reduction in Titers from Lentivirus Vectors Carrying Chromatin Insulator Elements in the 3′ LTR

Fabrizia Urbinati, Paritha Arumugam, Tomoyasu Higashimoto, Anil Perumbeti, Ping Xia, Punam Malik

Blood (2008) 112 (11): 2359.

https://doi.org/10.1182/blood.V112.11.2359.2359

Published: 2008

Journal Articles

Correction of Sickle Cell Anemia with γ-Globin Gene Delivered by Lentivirus Vector in the Setting of Myeloablative or Reduced Intensity Conditioning, and Establishing Critical Determinants for Successful Gene Therapy for Sickle Cell Disease.

Ajay Perumbeti, M.D., Tomoyasu Higashimoto, Phd., Fabrizia Urbinati, Phd., Kristy Lauderback, B.S., Anastacia Loberg, B.S., Gabriel Estevez-Pagani, Herbert Meiselman, M.D., Robert Franco, Phd., David Witte, M.D., Clinton H. Joiner, MD, PhD, Punam Malik

Blood (2008) 112 (11): 2354.

https://doi.org/10.1182/blood.V112.11.2354.2354

Published: 2008

Journal Articles

Elements of a Chromatin Insulator Necessary for Consistent, High-Level Globin Gene Expression

Paritha I. Arumugam, PhD, Ping Xia, MD, Fabrizia Urbinati, PhD, Punam Malik, MD

Blood (2007) 110 (11): 3731.

https://doi.org/10.1182/blood.V110.11.3731.3731

Published: 2007

Journal Articles

Mechanism of Reduction in Titers with Vectors Flanked by Chromatin Insulator Elements

Fabrizia Urbinati, PhD, Paritha Arumugam, Ph.D, Tomoyasu Higashimoto, Ph.D, Ping Xia, M.D, Punam Malik, M.D.

Blood (2007) 110 (11): 3734.

https://doi.org/10.1182/blood.V110.11.3734.3734

Published: 2007

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

Journal Articles

Molecular Follow-Up of Patients Treated with HSV-TK/ΔLNGFR Engineered Donor Lymphocytes after Allogeneic Stem Cell Transplantation.

Chiara Bonini, MD, Zulma Magnani, PhD, Alessandra Recchia, PhD, Fabrizia Urbinati, PhD, Sara Muraro, PhD, Serenella Sartori Tech, Fabio Ciceri, MD, Massimo Bernardi, MD, Alessandra Pescarollo, MD, Corrado Gallo-Stampino, MD, Marco Bregni, MD, Claudio Bordignon, MD, Fulvio Mavilio, PhD

Blood (2004) 104 (11): 1741.

https://doi.org/10.1182/blood.V104.11.1741.1741

Published: 2004

NARROW

Enrichment of Hematopoietic Stem Cells Using Immunomagnetic Beads to Facilitate Gene Therapy

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

Zinc Finger Nucleases Targeting The β-Globin Locus Drive Efficient Correction Of The Sickle Mutation In CD34 + Cells

Increasing Transduction Efficiency in Human Hematopoietic Stem Cells for Gene Therapy

Laser Tweezers Raman Spectroscopy As a Novel Red Blood Cell Functional Assay for Sickle Cell Disease

Preclinical Studies for Sickle Cell Disease Gene Therapy Using Bone Marrow CD34+ Cells Modified with a βAS3-Globin Lentiviral Vector

A Novel Combination of Chicken Hypersensitive Site-4 Insulator Elements Improves Titers and Restores Full Insulator Activity.

A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction

The 3′ End of the Chicken Hypersensitive Site-4 Insulator Has Properties Similar to the 5′ Insulator Core and Is Necessary in Conjunction with the Core for Full Insulator Activity

Mechanism of Reduction in Titers from Lentivirus Vectors Carrying Chromatin Insulator Elements in the 3′ LTR

Correction of Sickle Cell Anemia with γ-Globin Gene Delivered by Lentivirus Vector in the Setting of Myeloablative or Reduced Intensity Conditioning, and Establishing Critical Determinants for Successful Gene Therapy for Sickle Cell Disease.

Elements of a Chromatin Insulator Necessary for Consistent, High-Level Globin Gene Expression

Mechanism of Reduction in Titers with Vectors Flanked by Chromatin Insulator Elements

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.

Molecular Follow-Up of Patients Treated with HSV-TK/ΔLNGFR Engineered Donor Lymphocytes after Allogeneic Stem Cell Transplantation.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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