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Journal Articles

Taming the flame: WAS and IL-1 blockade

Sung-Yun Pai

Blood (2024) 144 (16): 1652–1653.

https://doi.org/10.1182/blood.2024026121

Published: 2024

Journal Articles

Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Jennifer W. Leiding, Danielle E. Arnold, Suhag Parikh, Brent Logan, Rebecca A. Marsh, Linda M. Griffith, Ruizhe Wu, Sharon Kidd, Kanwaldeep Mallhi, Deepak Chellapandian, Stephanie J. Si Lim, Eyal Grunebaum, E. Liana Falcone, Luis Murguia-Favela, Debbi Grossman, Vinod K. Prasad, Jennifer R. Heimall, Fabien Touzot, Lauri M. Burroughs, Jack Bleesing, Neena Kapoor, Jasmeen Dara, Olatundun Williams, Malika Kapadia, Benjamin R. Oshrine, Jeffrey J. Bednarski, Ahmad Rayes, Hey Chong, Geoffrey D. E. Cuvelier, Lisa R. Forbes Satter, Caridad Martinez, Mark T. Vander Lugt, Lolie C. Yu, Shanmuganathan Chandrakasan, Avni Joshi, Susan E. Prockop, Blachy J. Dávila Saldaña, Victor Aquino, Larisa A. Broglie, Christen L. Ebens, Lisa M. Madden, Kenneth DeSantes, Jordan Milner, Hemalatha G. Rangarajan, Ami J. Shah, Alfred P. Gillio, Alan P. Knutsen, Holly K. Miller, Theodore B. Moore, Pamela Graham, Andrea Bauchat, Nancy J. Bunin, Pierre Teira, Aleksandra Petrovic, Sharat Chandra, Hisham Abdel-Azim, Morna J. Dorsey, Olga Birbrayer, Morton J. Cowan, Christopher C. Dvorak, Elie Haddad, Donald B. Kohn, Luigi D. Notarangelo, Sung-Yun Pai, Jennifer M. Puck, Michael A. Pulsipher, Troy R. Torgerson, Harry L. Malech, Elizabeth M. Kang

Blood (2023) 142 (24): 2105–2118.

https://doi.org/10.1182/blood.2022019586

Published: 2023

Includes: Supplemental data

Journal Articles

Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome

Blood (2023) 142 (15): 1281–1296.

https://doi.org/10.1182/blood.2022019117

Published: 2023

Includes: Supplemental data

Journal Articles

Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion

Blood (2022) 140 (Supplement 1): 7770–7771.

https://doi.org/10.1182/blood-2022-159979

Published: 2022

Journal Articles

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Blood (2022) 140 (Supplement 1): 10643–10645.

https://doi.org/10.1182/blood-2022-159768

Published: 2022

Journal Articles

Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC

Geoffrey D. E. Cuvelier, Brent R. Logan, Susan E. Prockop, Rebecca H. Buckley, Caroline Y. Kuo, Linda M. Griffith, Xuerong Liu, Alison Yip, Michael S. Hershfield, Paul G. Ayoub, Theodore B. Moore, Morna J. Dorsey, Richard J. O’Reilly, Neena Kapoor, Sung-Yun Pai, Malika Kapadia, Christen L. Ebens, Lisa R. Forbes Satter, Lauri M. Burroughs, Aleksandra Petrovic, Deepak Chellapandian, Jennifer Heimall, David C. Shyr, Ahmad Rayes, Jeffrey J. Bednarski, Sharat Chandra, Shanmuganathan Chandrakasan, Alfred P. Gillio, Lisa Madden, Troy C. Quigg, Emi H. Caywood, Blachy J. Dávila Saldaña, Kenneth DeSantes, Hesham Eissa, Frederick D. Goldman, Jacob Rozmus, Ami J. Shah, Mark T. Vander Lugt, Monica S. Thakar, Roberta E. Parrott, Caridad Martinez, Jennifer W. Leiding, Troy R. Torgerson, Michael A. Pulsipher, Luigi D. Notarangelo, Morton J. Cowan, Christopher C. Dvorak, Elie Haddad, Jennifer M. Puck, Donald B. Kohn

Blood (2022) 140 (7): 685–705.

https://doi.org/10.1182/blood.2022016196

Published: 2022

Includes: Supplemental data

Journal Articles

Peri-Transplant Alemtuzumab Levels Predict Risk of Secondary Graft Failure and Inversely Impact CXCL9 Levels after RIC HCT (A Correlative Biology Study to BMT-CTN 1204 RICHI)

Ashley V Geerlinks, Brooks Scull, Christa Krupski, Ryan Fleischmann, Michael A. Pulsipher, Mary Eapen, Jim A. Connelly, Catherine M. Bollard, Sung-Yun Pai, Christine N. Duncan, Leslie S Kean, K. Scott Baker, Lauri S Burroughs, Jeffrey R. Andolina, Michael B Jordan, Tsz-Kwong Man, Carl E. Allen, Rebecca A. Marsh

Blood (2021) 138 (Supplement 1): 748.

https://doi.org/10.1182/blood-2021-145629

Published: 2021

Journal Articles

Built to last: gene therapy for ADA SCID

Sung-Yun Pai

Blood (2021) 138 (15): 1287–1288.

https://doi.org/10.1182/blood.2021012300

Published: 2021

Journal Articles

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Lauri M. Burroughs, Aleksandra Petrovic, Ruta Brazauskas, Xuerong Liu, Linda M. Griffith, Hans D. Ochs, Jack J. Bleesing, Stephanie Edwards, Christopher C. Dvorak, Sonali Chaudhury, Susan E. Prockop, Ralph Quinones, Frederick D. Goldman, Troy C. Quigg, Shanmuganathan Chandrakasan, Angela R. Smith, Suhag Parikh, Blachy J. Dávila Saldaña, Monica S. Thakar, Rachel Phelan, Shalini Shenoy, Lisa R. Forbes, Caridad Martinez, Deepak Chellapandian, Evan Shereck, Holly K. Miller, Neena Kapoor, Jessie L. Barnum, Hey Chong, David C. Shyr, Karin Chen, Rolla Abu-Arja, Ami J. Shah, Katja G. Weinacht, Theodore B. Moore, Avni Joshi, Kenneth B. DeSantes, Alfred P. Gillio, Geoffrey D. E. Cuvelier, Michael D. Keller, Jacob Rozmus, Troy Torgerson, Michael A. Pulsipher, Elie Haddad, Kathleen E. Sullivan, Brent R. Logan, Donald B. Kohn, Jennifer M. Puck, Luigi D. Notarangelo, Sung-Yun Pai, David J. Rawlings, Morton J. Cowan

Blood (2020) 135 (23): 2094–2105.

https://doi.org/10.1182/blood.2019002939

Published: 2020

Journal Articles

Inducible Phase Separation of GSK3α As a Mechanism for Asparaginase Resistance in Acute Leukemias

Laura Hinze, Sabine Schreek, Andre Zeug, PhD, Evgeni Ponimaskin, PhD, Roxane Labrosse, MD FRCPC, James Degar, Gabriela Chiosis, PhD, Sung-Yun Pai, MD, Kristen E. Stevenson, MS, Donna S Neuberg, ScD, Beat Bornhauser, PhD, Jean-Pierre Bourquin, MD PhD, Martin Stanulla, MD, Alejandro Gutierrez, MD

Blood (2019) 134 (Supplement_1): 169.

https://doi.org/10.1182/blood-2019-126633

Published: 2019

Journal Articles

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Blood (2019) 134 (Supplement_1): 4629.

https://doi.org/10.1182/blood-2019-126161

Published: 2019

Journal Articles

SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery

Elie Haddad, Brent R. Logan, Linda M. Griffith, Rebecca H. Buckley, Roberta E. Parrott, Susan E. Prockop, Trudy N. Small, Jessica Chaisson, Christopher C. Dvorak, Megan Murnane, Neena Kapoor, Hisham Abdel-Azim, Imelda C. Hanson, Caridad Martinez, Jack J. H. Bleesing, Sharat Chandra, Angela R. Smith, Matthew E. Cavanaugh, Soma Jyonouchi, Kathleen E. Sullivan, Lauri Burroughs, Suzanne Skoda-Smith, Ann E. Haight, Audrey G. Tumlin, Troy C. Quigg, Candace Taylor, Blachy J. Dávila Saldaña, Michael D. Keller, Christine M. Seroogy, Kenneth B. Desantes, Aleksandra Petrovic, Jennifer W. Leiding, David C. Shyr, Hélène Decaluwe, Pierre Teira, Alfred P. Gillio, Alan P. Knutsen, Theodore B. Moore, Morris Kletzel, John A. Craddock, Victor Aquino, Jeffrey H. Davis, Lolie C. Yu, Geoffrey D. E. Cuvelier, Jeffrey J. Bednarski, Frederick D. Goldman, Elizabeth M. Kang, Evan Shereck, Matthew H. Porteus, James A. Connelly, Thomas A. Fleisher, Harry L. Malech, William T. Shearer, Paul Szabolcs, Monica S. Thakar, Mark T. Vander Lugt, Jennifer Heimall, Ziyan Yin, Michael A. Pulsipher, Sung-Yun Pai, Donald B. Kohn, Jennifer M. Puck, Morton J. Cowan, Richard J. O'Reilly, Luigi D. Notarangelo

Blood (2018) 132 (17): 1737–1749.

https://doi.org/10.1182/blood-2018-03-840702

Published: 2018

Includes: Supplemental data

Journal Articles

Reduced-intensity conditioning for hematopoietic cell transplant for HLH and primary immune deficiencies

Blood (2018) 132 (13): 1438–1451.

https://doi.org/10.1182/blood-2018-01-828277

Published: 2018

Includes: Supplemental data

Journal Articles

B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation

Blood (2018) 131 (26): 2967–2977.

https://doi.org/10.1182/blood-2017-10-809822

Published: 2018

Includes: Supplemental data

Journal Articles

Patients with CD3G mutations reveal a role for human CD3γ in T reg diversity and suppressive function

Jared H. Rowe, Ottavia M. Delmonte, Sevgi Keles, Brian D. Stadinski, Adam K. Dobbs, Lauren A. Henderson, Yasuhiro Yamazaki, Luis M. Allende, Francisco A. Bonilla, Luis I. Gonzalez-Granado, Seyma Celikbilek Celik, Sukru N. Guner, Hasan Kapakli, Christina Yee, Sung-Yun Pai, Eric S. Huseby, Ismail Reisli, Jose R. Regueiro, Luigi D. Notarangelo

Blood (2018) 131 (21): 2335–2344.

https://doi.org/10.1182/blood-2018-02-835561

Published: 2018

Includes: Supplemental data

Journal Articles

Immune reconstitution and survival of 100 SCID patients post–hematopoietic cell transplant: a PIDTC natural history study

Blood (2017) 130 (25): 2718–2727.

https://doi.org/10.1182/blood-2017-05-781849

Published: 2017

Includes: Supplemental data

Journal Articles

Use of Eculizumab in a Patient with Refractory Autoimmune Hemolytic Anemia with Heterozygous NFκB1 Mutation

Katharine Halligan, MD PhD, Lana Vasiljevic, Phillip Roehrs, MD, Andrew Mcginnitie, MD, Sung-Yun Pai, MD, Rachael F. Grace, MD, Maria Olivares Boucher, MD, Lauren Weintraub, MD

Blood (2017) 130 (Supplement 1): 4743.

https://doi.org/10.1182/blood.V130.Suppl_1.4743.4743

Published: 2017

Journal Articles

Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome

Blood (2017) 129 (21): 2928–2938.

https://doi.org/10.1182/blood-2016-11-745638

Published: 2017

Includes: Supplemental data

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

Stem cell transplantation for tetratricopeptide repeat domain 7A deficiency: long-term follow-up

Jochen Kammermeier, Giovanna Lucchini, Sung-Yun Pai, Austen Worth, Dyanne Rampling, Persis Amrolia, Juliana Silva, Robert Chiesa, Kanchan Rao, Gabriele Noble-Jamieson, Marco Gasparetto, Drew Ellershaw, Holm Uhlig, Neil Sebire, Mamoun Elawad, Luigi Notarangelo, Neil Shah, Paul Veys

Blood (2016) 128 (9): 1306–1308.

https://doi.org/10.1182/blood-2016-01-696385

Published: 2016

Includes: Supplemental data

NARROW

Taming the flame: WAS and IL-1 blockade

Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome

Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC

Peri-Transplant Alemtuzumab Levels Predict Risk of Secondary Graft Failure and Inversely Impact CXCL9 Levels after RIC HCT (A Correlative Biology Study to BMT-CTN 1204 RICHI)

Built to last: gene therapy for ADA SCID

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Inducible Phase Separation of GSK3α As a Mechanism for Asparaginase Resistance in Acute Leukemias

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery

Reduced-intensity conditioning for hematopoietic cell transplant for HLH and primary immune deficiencies

B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation

Patients with CD3G mutations reveal a role for human CD3γ in T reg diversity and suppressive function

Immune reconstitution and survival of 100 SCID patients post–hematopoietic cell transplant: a PIDTC natural history study

Use of Eculizumab in a Patient with Refractory Autoimmune Hemolytic Anemia with Heterozygous NFκB1 Mutation

Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome

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

Stem cell transplantation for tetratricopeptide repeat domain 7A deficiency: long-term follow-up

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American Society of Hematology

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American Society of Hematology

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