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Journal Articles

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Blood (2019) 134 (Supplement_1): 608.

https://doi.org/10.1182/blood-2019-127439

Published: 2019

Journal Articles

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study

Blood (2019) 134 (Supplement_1): 2058.

https://doi.org/10.1182/blood-2019-126746

Published: 2019

Journal Articles

Interim Results from a Phase I/II Clinical Gene Therapy Study for Newly Diagnosed Infants with X-Linked Severe Combined Immunodeficiency Using a Safety-Modified Lentiviral Vector and Targeted Reduced Exposure to Busulfan

Blood (2017) 130 (Supplement 1): 523.

https://doi.org/10.1182/blood.V130.Suppl_1.523.523

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

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

Lentiviral Hematopoietic Stem Cell Gene Therapy for Older Patients with X-Linked Severe Combined Immunodeficiency

Suk See De Ravin, MDPhD, Xiaolin Wu, Narda Theobald, Janet S Lee, John Gray, PhD, Imelda Celine Hanson, MD, Luigi Notarangelo, MDPhD, Brian P. Sorrentino, MD, Harry L. Malech, MD

Blood (2015) 126 (23): 261.

https://doi.org/10.1182/blood.V126.23.261.261

Published: 2015

Journal Articles

Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector

Blood (2013) 122 (21): 715.

https://doi.org/10.1182/blood.V122.21.715.715

Published: 2013

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

Transduction of Human CD34+ Derived NSG Repopulating Cells with An Insulated SIN Lentiviral Vector for SCID-X1 From a Stable Producer Cell Line At Clinical Scale

Michael R. Greene, MD, Timothy Lockey, PhD, John T. Gray, PhD, Paul W. Eldridge, PhD, Yoon-Sang Kim, MD, PhD, Perdeep K. Mehta, PhD, Brian P. Sorrentino, MD

Blood (2011) 118 (21): 670.

https://doi.org/10.1182/blood.V118.21.670.670

Published: 2011

Journal Articles

Effects of LMO2 on Human T-Cell Development Are Modulated by Notch Signaling

James A Kennedy, MD, PhD, Renata Teixeira, Sara Berthiaume, Frederic Barabe, MD

Blood (2011) 118 (21): 2470.

https://doi.org/10.1182/blood.V118.21.2470.2470

Published: 2011

Journal Articles

Integration-Mediated Activation of PRDM16 and HMGA2 in Multiple Clones without Adverse Hematopoietic Consequences Following Transplant of Autologous MGMTP140K Gene-Modified CD34 + Cells

Jennifer E Adair, PhD, Brian C Beard, PhD, Grant Trobridge, PhD, Frederic Bushman, PhD, Maciej M Mrugala, MD PhD, Hans-Peter Kiem, MD

Blood (2011) 118 (21): 2053.

https://doi.org/10.1182/blood.V118.21.2053.2053

Published: 2011

Journal Articles

The Yin and Yang of Interleukin-21 in Allergy, Autoimmunity and Cancer

Warren J. Leonard, MD

Blood (2011) 118 (21): SCI-6.

https://doi.org/10.1182/blood.V118.21.SCI-6.SCI-6

Published: 2011

Journal Articles

Successful Reduced-Intensity Stem Cell Transplantation From Unrelated Cord Blood In Three Patients with X-Linked Severe Combined Immunodeficiency.

Akihiro Iguchi, MD, PhD, Mizuho Ichikawa, MD, PhD, Nobuaki Kawamura, MD, PhD, Shunichiro Takezaki, MD, Yuka Okura, MD, Jun Inamoto, MD, Junjiro Ohshima, MD, PhD, Tomonobu Sato, MD, PhD, Masahide Nakajima, MD, PhD, Makoto Kaneda, MD, Yuko Cho, MD, PhD, Masafumi Yamada, MD, PhD, Ryoji Kobayashi, MD, PhD, Ichiro Kobayashi, MD, PhD, Tadashi Ariga, MD, PhD

Blood (2010) 116 (21): 4571.

https://doi.org/10.1182/blood.V116.21.4571.4571

Published: 2010

Journal Articles

Modeling Human T Cell Acute Lymphoblastic Leukemia In Vitro and In Vivo with LMO2.

James A Kennedy, Sara Berthiaume, Frederic Barabe, MD

Blood (2010) 116 (21): 3648.

https://doi.org/10.1182/blood.V116.21.3648.3648

Published: 2010

Journal Articles

Clinical and immunologic consequences of a somatic reversion in a patient with X-linked severe combined immunodeficiency

Carsten Speckmann, Ulrich Pannicke, Elisabeth Wiech, Klaus Schwarz, Paul Fisch, Wilhelm Friedrich, Tim Niehues, Kimberly Gilmour, Karin Buiting, Michael Schlesier, Hermann Eibel, Jan Rohr, Andrea Superti-Furga, Ute Groß-Wieltsch, Stephan Ehl

Blood (2008) 112 (10): 4090–4097.

https://doi.org/10.1182/blood-2008-04-153361

Published: 2008

Journal Articles

Detection of T lymphocytes with a second-site mutation in skin lesions of atypical X-linked severe combined immunodeficiency mimicking Omenn syndrome

Taizo Wada, Masahiro Yasui, Tomoko Toma, Yuko Nakayama, Mika Nishida, Masaki Shimizu, Michiko Okajima, Yoshihito Kasahara, Shoichi Koizumi, Masami Inoue, Keisei Kawa, Akihiro Yachie

Blood (2008) 112 (5): 1872–1875.

https://doi.org/10.1182/blood-2008-04-149708

Published: 2008

Journal Articles

An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation

Byoung Y. Ryu, Marguerite V. Evans-Galea, John T. Gray, David M. Bodine, Derek A. Persons, Arthur W. Nienhuis

Blood (2008) 111 (4): 1866–1875.

https://doi.org/10.1182/blood-2007-04-085506

Published: 2008

Includes: Supplemental data

Journal Articles

Reduced Genotoxic Risk Using Foamy Viral Vectors for the Treatment of Canine Leukocyte Adhesion Deficiency

Thomas R. Bauer, Jr, PhD, Mehreen Hai, Ph.D., Rima L. Adler, Ph.D., James M. Allen, Ph.D., Laura M. Tuschong, B.S., Erik M. Olson, B.S., Dennis D. Hickstein, M.D., David W. Russell, M.D., Ph.D.

Blood (2007) 110 (11): 3744.

https://doi.org/10.1182/blood.V110.11.3744.3744

Published: 2007

Journal Articles

Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells

Fang Zhang, Susannah I. Thornhill, Steven J. Howe, Meera Ulaganathan, Axel Schambach, Joanna Sinclair, Christine Kinnon, H. Bobby Gaspar, Michael Antoniou, Adrian J. Thrasher

Blood (2007) 110 (5): 1448–1457.

https://doi.org/10.1182/blood-2006-12-060814

Published: 2007

Includes: Supplemental data

Journal Articles

Unique Integration Profiles of Gammaretrovirus, Lentivirus, and Foamy Virus Transduced Dog Long-Term Repopulating Cells.

Kirsten A. Keyser, Brian C. Beard, PhD, Grant Trobridge, PhD, Laura J. Peterson, Dan Miller, PhD, Mike Jacobs, PhD, Rajinder Kaul, PhD, Hans-Peter Kiem, MD

Blood (2006) 108 (11): 3252.

https://doi.org/10.1182/blood.V108.11.3252.3252

Published: 2006

NARROW

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study

Interim Results from a Phase I/II Clinical Gene Therapy Study for Newly Diagnosed Infants with X-Linked Severe Combined Immunodeficiency Using a Safety-Modified Lentiviral Vector and Targeted Reduced Exposure to Busulfan

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

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

Lentiviral Hematopoietic Stem Cell Gene Therapy for Older Patients with X-Linked Severe Combined Immunodeficiency

Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector

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

Transduction of Human CD34+ Derived NSG Repopulating Cells with An Insulated SIN Lentiviral Vector for SCID-X1 From a Stable Producer Cell Line At Clinical Scale

Effects of LMO2 on Human T-Cell Development Are Modulated by Notch Signaling

Integration-Mediated Activation of PRDM16 and HMGA2 in Multiple Clones without Adverse Hematopoietic Consequences Following Transplant of Autologous MGMTP140K Gene-Modified CD34 + Cells

The Yin and Yang of Interleukin-21 in Allergy, Autoimmunity and Cancer

Successful Reduced-Intensity Stem Cell Transplantation From Unrelated Cord Blood In Three Patients with X-Linked Severe Combined Immunodeficiency.

Modeling Human T Cell Acute Lymphoblastic Leukemia In Vitro and In Vivo with LMO2.

Clinical and immunologic consequences of a somatic reversion in a patient with X-linked severe combined immunodeficiency

Detection of T lymphocytes with a second-site mutation in skin lesions of atypical X-linked severe combined immunodeficiency mimicking Omenn syndrome

An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation

Reduced Genotoxic Risk Using Foamy Viral Vectors for the Treatment of Canine Leukocyte Adhesion Deficiency

Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells

Unique Integration Profiles of Gammaretrovirus, Lentivirus, and Foamy Virus Transduced Dog Long-Term Repopulating Cells.

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American Society of Hematology

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American Society of Hematology

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