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Journal Articles

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency

Blood (2021) 138 (15): 1304–1316.

https://doi.org/10.1182/blood.2020010260

Published: 2021

Includes: Supplemental data

Journal Articles

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Blood (2019) 134 (Supplement_1): 3345.

https://doi.org/10.1182/blood-2019-123432

Published: 2019

Journal Articles

Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients

Aaron R. Cooper, Georgia R. Lill, Kit Shaw, Denise A. Carbonaro-Sarracino, Alejandra Davila, Robert Sokolic, Fabio Candotti, Matteo Pellegrini, Donald B. Kohn

Blood (2017) 129 (19): 2624–2635.

https://doi.org/10.1182/blood-2016-12-756734

Published: 2017

Includes: Supplemental data

Journal Articles

Wiskott-Aldrich Syndrome: A Retrospective Study on 575 Patients Analyzing the Impact of Splenectomy, Stem Cell Transplantation, or No Definitive Treatment on Frequency of Disease-Related Complications and Physician-Perceived Quality of Life

Jannik S Glasmacher, Tanja C Bittner, MD, Hans D Ochs, MD, Alessandro Aiuti, MD PhD, Peter D Arkwright, MD, Dmitry Balashov, Uta Behrends, Bernd H. Belohradsky, MD, Elisa Bertoni, David K. Buchbinder, MD MSHS, Michael Browning, Anastasiia Bondarenko, Fabio Candotti, MD, Alessandro Cattoni, Liudmyla Chernyshova, Joseph H. Chewning, MD, Peter Ciznar, Theresa Cole, Beatriz T Costa-Carvalho, Wojciech Czogala, Gregor Dueckers, David M Edgar, Fatih Erbey, Anders Fasth, MD PhD, Renata Formankova, MD PhD, Tomas Freiberger, Eleonora Gambineri, Andrew Gennery, MD, Frederick D Goldman, MD, Luis I Gonzalez-Granado, Tiago N Gulmaraes, David Hagin, MD PhD, Fabian Hauck, MD PhD, Tarja Heiskanen-Kosma, Manfred Hoenig, MD, Hanna Juntti, Hirokazu Kanegane, Leena Kainulainen, Neslihan E Karaca, Sara S Kilic, Christoph Klein, MD PhD, Sylwia Koltan, Irina Kondratenko, Dawei Liu, MD, Susanne Matthes, MD, Juliana T L Mazzucchelli, Isabelle Meyts, Siraj Misbah, Zohreh Nademi, Gulnara Nasrullayeva, Lucia D Notarangelo, Pere Soler-Palacin, Olga Pashchenko, Srdjan Pasic, Isabelle Pellier, MD, Claudio Pignata, Camilla Roepstorff, Catharina Schuetz, Ansgar S Schulz, MD PhD Prof., Gesmar R S Segundo, Anna Shcherbina, Joanne Smart, Robert A. Sokolic, MD, Polina Stepensky, MD, Troy Torgerson, MD PhD, Svetlana Vakhlyarskaya, Joris van Montfrans, MD PhD, Kim Vettenranta, MD PD, Beata Wolska-Kusnierz, Xiaodong Zhao, John B Ziegler, Xuan Zhang, Michael H. Albert, MD

Blood (2016) 128 (22): 366.

https://doi.org/10.1182/blood.V128.22.366.366

Published: 2016

Journal Articles

N-WASP is required for B-cell–mediated autoimmunity in Wiskott-Aldrich syndrome

Stefano Volpi, Elettra Santori, Katrina Abernethy, Masayuki Mizui, Carin I. M. Dahlberg, Mike Recher, Kelly Capuder, Eva Csizmadia, Douglas Ryan, Divij Mathew, George C. Tsokos, Scott Snapper, Lisa S. Westerberg, Adrian J. Thrasher, Fabio Candotti, Luigi D. Notarangelo

Blood (2016) 127 (2): 216–220.

https://doi.org/10.1182/blood-2015-05-643817

Published: 2016

Includes: Supplemental data

Journal Articles

What's the 'Skinny' on Microbiome? Interplay of Immune Cells, Microbes, and Skin Barrier in Health and Disease

Julie Segre, PhD, Heidi Kong, MD, Fabio Candotti, MD, Steven M. Holland, MD, Alexandra F Freeman, MD, Julia Oh, PhD, Robert A. Sokolic

Blood (2014) 124 (21): SCI-46.

https://doi.org/10.1182/blood.V124.21.SCI-46.SCI-46

Published: 2014

Journal Articles

Cation Leak in Red Blood Cells of Patients with Wiskott-Aldrich Syndrome Leads to Non-Immunologic Hemolysis

Robert A. Sokolic, MD, Satheesh Chonat, MD, Mary Risinger, RN, Michael Eckhaus, DVM, Elizabeth Garabedian, RN, MSLS, Katherine R. Calvo, MD, PhD, Irina Maric, MD, Theodosia A. Kalfa, MD PhD, Fabio Candotti, MD

Blood (2014) 124 (21): 1338.

https://doi.org/10.1182/blood.V124.21.1338.1338

Published: 2014

Journal Articles

AK2 Deficiency In Zebrafish Recapitulates Human Reticular Dysgenesis, An Autosomal Recessive Form Of Severe Combined Immunodeficiency

Alberto Rissone, PhD, Jaya Jagadeesh, Karen Simon, Kevin Bishop, Raman B. Sood, PhD, Fabio Candotti, MD

Blood (2013) 122 (21): 2416.

https://doi.org/10.1182/blood.V122.21.2416.2416

Published: 2013

Journal Articles

Negative Control Region Is a Critical Element Of Insertional Oncogenesis After Gene Transfer Into Hematopoietic Progenitors With Moloney Murine Leukemia Viruses

Yasuhiro Ikawa, MD, PhD, Toru Uchiyama, MD, PhD, Guridevi Jayashree Jagadeesh, MS, Fabio Candotti, MD

Blood (2013) 122 (21): 164.

https://doi.org/10.1182/blood.V122.21.164.164

Published: 2013

Journal Articles

Adenylate Kinase 2 Regulates Zebrafish Primitive and Definitive Hematopoiesis

Alberto Rissone, PhD, Jaya Jagadeesh, Simon Karen, Kevin Bishop, Trevor Blake, Raman B. Sood, PhD, Fabio Candotti, MD

Blood (2012) 120 (21): 1208.

https://doi.org/10.1182/blood.V120.21.1208.1208

Published: 2012

Journal Articles

Analysis of Risk and Mechanism of Insertional Oncogenesis After Gene Transfer Into Hematopoietic Progenitors with Integrating Viral Vectors

Yasuhiro Ikawa, MD, PhD, Toru Uchiyama, MD, PhD, Guridevi Jayashree Jagadeesh, MS, Fabio Candotti, MD

Blood (2012) 120 (21): 2049.

https://doi.org/10.1182/blood.V120.21.2049.2049

Published: 2012

Journal Articles

Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans

Blood (2012) 120 (18): 3635–3646.

https://doi.org/10.1182/blood-2012-02-400937

Published: 2012

Includes: Supplemental data

Journal Articles

B cell–intrinsic deficiency of the Wiskott-Aldrich syndrome protein (WASp) causes severe abnormalities of the peripheral B-cell compartment in mice

Blood (2012) 119 (12): 2819–2828.

https://doi.org/10.1182/blood-2011-09-379412

Published: 2012

Includes: Supplemental data

Journal Articles

Platelets From WAS Patients Are More Susceptible Than Controls to Phagocytosis by Activated THP-1 Cells

Ted Strom, MD, PhD, Xueying Zeng, Fabio Candotti, MD, PhD, Mary Ellen Conley, MD, PhD, Praveen Anur, MD, Amanda Prislovsky

Blood (2011) 118 (21): 2222.

https://doi.org/10.1182/blood.V118.21.2222.2222

Published: 2011

Journal Articles

Characterization of AK2 Gene Function in Zebrafish Hematopoiesis

Alberto F Rissone, Guridevi Jayashree Jagadeesh, MS, Karen Simon, Kevin Bishop, Milton English, Trevor Blake, Robert M Nissen, Raman B. Sood, PhD, Fabio Candotti, MD

Blood (2011) 118 (21): 2185.

https://doi.org/10.1182/blood.V118.21.2185.2185

Published: 2011

Journal Articles

Comparison of Immortalization Potential of Gamma-Retroviral, Lentiviral and Foamy Virus Gene Transfer Vectors

Yasuhiro Ikawa, MD, PhD, Toru Uchiyama, MD, PhD, Guridevi Jayashree Jagadeesh, MS, Fabio Candotti, MD

Blood (2011) 118 (21): 3116.

https://doi.org/10.1182/blood.V118.21.3116.3116

Published: 2011

Journal Articles

Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency

Robert Sokolic, Irina Maric, Chimene Kesserwan, Elizabeth Garabedian, I. Celine Hanson, Margaret Dodds, Rebecca Buckley, Andrew C. Issekutz, Naynesh Kamani, Kit Shaw, Ben Tan, Pawan Bali, Michael S. Hershfield, Donald B. Kohn, Alan S. Wayne, Fabio Candotti

Blood (2011) 118 (10): 2688–2694.

https://doi.org/10.1182/blood-2011-01-329359

Published: 2011

Journal Articles

Defective inhibition of B-cell proliferation by Wiskott-Aldrich syndrome protein-deficient regulatory T cells

Marsilio Adriani, Krysten A. Jones, Toru Uchiyama, Martha R. Kirby, Christopher Silvin, Stacie M. Anderson, Fabio Candotti

Blood (2011) 117 (24): 6608–6611.

https://doi.org/10.1182/blood-2010-12-322834

Published: 2011

Includes: Supplemental data

Journal Articles

Systemic autoimmunity and defective Fas ligand secretion in the absence of the Wiskott-Aldrich syndrome protein

Nikolay P. Nikolov, Masaki Shimizu, Sophia Cleland, Daniel Bailey, Joseph Aoki, Ted Strom, Pamela L. Schwartzberg, Fabio Candotti, Richard M. Siegel

Blood (2010) 116 (5): 740–747.

https://doi.org/10.1182/blood-2009-08-237560

Published: 2010

Includes: Supplemental data

Journal Articles

Reduced Number of Dense Bodies and Reduced Serotonin Content in Platelets of Patients with Wiskott-Aldrich Syndrome.

Robert A. Sokolic, Thomas Markello, Marcy Krumwiede, Roxanne Fischer, Elizabeth Garabedian, William A. Gahl, James G. White, Fabio Candotti

Blood (2009) 114 (22): 1321.

https://doi.org/10.1182/blood.V114.22.1321.1321

Published: 2009

NARROW

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients

Wiskott-Aldrich Syndrome: A Retrospective Study on 575 Patients Analyzing the Impact of Splenectomy, Stem Cell Transplantation, or No Definitive Treatment on Frequency of Disease-Related Complications and Physician-Perceived Quality of Life

N-WASP is required for B-cell–mediated autoimmunity in Wiskott-Aldrich syndrome

What's the 'Skinny' on Microbiome? Interplay of Immune Cells, Microbes, and Skin Barrier in Health and Disease

Cation Leak in Red Blood Cells of Patients with Wiskott-Aldrich Syndrome Leads to Non-Immunologic Hemolysis

AK2 Deficiency In Zebrafish Recapitulates Human Reticular Dysgenesis, An Autosomal Recessive Form Of Severe Combined Immunodeficiency

Negative Control Region Is a Critical Element Of Insertional Oncogenesis After Gene Transfer Into Hematopoietic Progenitors With Moloney Murine Leukemia Viruses

Adenylate Kinase 2 Regulates Zebrafish Primitive and Definitive Hematopoiesis

Analysis of Risk and Mechanism of Insertional Oncogenesis After Gene Transfer Into Hematopoietic Progenitors with Integrating Viral Vectors

Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans

B cell–intrinsic deficiency of the Wiskott-Aldrich syndrome protein (WASp) causes severe abnormalities of the peripheral B-cell compartment in mice

Platelets From WAS Patients Are More Susceptible Than Controls to Phagocytosis by Activated THP-1 Cells

Characterization of AK2 Gene Function in Zebrafish Hematopoiesis

Comparison of Immortalization Potential of Gamma-Retroviral, Lentiviral and Foamy Virus Gene Transfer Vectors

Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency

Defective inhibition of B-cell proliferation by Wiskott-Aldrich syndrome protein-deficient regulatory T cells

Systemic autoimmunity and defective Fas ligand secretion in the absence of the Wiskott-Aldrich syndrome protein

Reduced Number of Dense Bodies and Reduced Serotonin Content in Platelets of Patients with Wiskott-Aldrich Syndrome.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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