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Frederick D. Goldman
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Journal Articles

Wiskott-Aldrich syndrome: a study of 577 patients defines the genotype as a biomarker for disease severity and survival

Clinical Trials & Observations
Tanja C. Vallée, Jannik S. Glasmacher, Hannes Buchner, Peter D. Arkwright, Uta Behrends, Anastasia Bondarenko, Michael J. Browning, David Buchbinder, Alessandro Cattoni, Liudmyla Chernyshova, Peter Ciznar, Theresa Cole, Wojciech Czogała, Gregor Dueckers, John David M. Edgar, Fatih Erbey, Anders Fasth, Francesca Ferrua, Renata Formankova, Eleonora Gambineri, Andrew R. Gennery, Frederick D. Goldman, Luis I. Gonzalez-Granado, Carsten Heilmann, Tarja Heiskanen-Kosma, Hanna Juntti, Leena Kainulainen, Hirokazu Kanegane, Neslihan E. Karaca, Sara S. Kilic, Christoph Klein, Sylwia Kołtan, Irina Kondratenko, Isabelle Meyts, Gulnara M. Nasrullayeva, Lucia D. Notarangelo, Srdjan Pasic, Isabelle Pellier, Claudio Pignata, Siraj Misbah, Ansgar Schulz, Gesmar R. Segundo, Anna Shcherbina, Mary Slatter, Robert Sokolic, Pere Soler-Palacin, Polina Stepensky, Joris M. van Montfrans, Samppa Ryhänen, Beata Wolska-Kuśnierz, John B. Ziegler, Xiaodong Zhao, Alessandro Aiuti, Hans D. Ochs, Michael H. Albert
Journal: Blood
Blood (2024) 143 (24): 2504–2516.
https://doi.org/10.1182/blood.2023021411
Published: 2024
Includes: Supplemental data
Journal Articles

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

CME Clinical Trials & Observations
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
Journal: Blood
Blood (2022) 140 (7): 685–705.
https://doi.org/10.1182/blood.2022016196
Published: 2022
Includes: Supplemental data
Journal Articles

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

Clinical Trials & Observations
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
Journal: Blood
Blood (2020) 135 (23): 2094–2105.
https://doi.org/10.1182/blood.2019002939
Published: 2020
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
Journal: Blood
Blood (2018) 132 (17): 1737–1749.
https://doi.org/10.1182/blood-2018-03-840702
Published: 2018
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
Journal: Blood
Blood (2016) 128 (22): 366.
https://doi.org/10.1182/blood.V128.22.366.366
Published: 2016
Journal Articles

Suppression of Antioxidant Responses in Dyskeratosis Congenita Cells

Erik Westin, PhD, Larisa Pereboeva, MD PhD, Divya Devadasan, MS, Tim M. Townes, PhD, Frederick D Goldman, MD
Journal: Blood
Blood (2015) 126 (23): 2412.
https://doi.org/10.1182/blood.V126.23.2412.2412
Published: 2015
Journal Articles

Consistent Upregulation of Oxidative Stress and DNA Damage Amongst Dyskeratosis Congenita Genotypes: Ameliorative Effects of Antioxidants

Larisa Pereboeva, MD PhD, Erik Westin, PhD, Meredith Hubbard, BS, Frederick D Goldman, MD
Journal: Blood
Blood (2014) 124 (21): 1603.
https://doi.org/10.1182/blood.V124.21.1603.1603
Published: 2014
Journal Articles

Crispr Modification of Induced Pluripotent Stem Derived from Diamond-Blackfan Anemia Fibroblasts

Erik Westin, PhD, Divya Devadasan, MA, Chao Li, Lei Ding, PhD, Tim M. Townes, PhD, Frederick D Goldman, MD
Journal: Blood
Blood (2014) 124 (21): 4804.
https://doi.org/10.1182/blood.V124.21.4804.4804
Published: 2014
Journal Articles

DNA Damage Responses and Oxidative Stress in Dyskeratosis Congenita.

Larisa Pereboeva, MD, PhD, Erik Westin, PhD, Toral Patel, Ian Flaniken, Lawrence S. Lamb, Jr., PhD, Aloysius Klingelhutz, PhD, Frederick D Goldman, MD
Journal: Blood
Blood (2012) 120 (21): 2359.
https://doi.org/10.1182/blood.V120.21.2359.2359
Published: 2012
Journal Articles

Telomere Elongation in Dyskeratosis Congenita Induced Pluripotent Stem Cells.

Suneet Agarwal, M.D., Ph.D., Yuin-Han Loh, Ph.D., Erin M McLoughlin, Junjiu Huang, In-Hyun Park, Ph.D., Justine D Miller, Hongguang Huo, Maja Okuka, Rosana Maria dos Reis, Sabine Loewer, Ph.D., David Keefe, M.D., Frederick D Goldman, M.D., Aloysius Klingelhutz, Ph.D., Lin Liu, Ph.D., George Q. Daley, M.D., Ph.D.
Journal: Blood
Blood (2009) 114 (22): 497.
https://doi.org/10.1182/blood.V114.22.497.497
Published: 2009
Journal Articles

Exogenous Immunoglobulin Downregulates T Cell Receptor Signaling and Cytokine Production

Daniel S Tawfi, BS, Wendy S Hamilton, BS, Katelyn R Cowan, BS, Frederick D Goldman, MD
Journal: Blood
Blood (2008) 112 (11): 2559.
https://doi.org/10.1182/blood.V112.11.2559.2559
Published: 2008
Journal Articles

Transduction of hTR into Hematopoietic Stem Cells Increases Telomerase Activity

Frederick D Goldman, Katelyn R Cowan, Daniel S Tawfik, Wendy Hamilton, Erik Westin, Aloysius Klingelhutz
Journal: Blood
Blood (2008) 112 (11): 4624.
https://doi.org/10.1182/blood.V112.11.4624.4624
Published: 2008
Journal Articles

Characterization of primitive hematopoietic cells from patients with dyskeratosis congenita

Frederick D. Goldman, Geraldine Aubert, Al J. Klingelhutz, Mark Hills, Sarah R. Cooper, Wendy S. Hamilton, Annette J. Schlueter, Karen Lambie, Connie J. Eaves, Peter M. Lansdorp
Journal: Blood
Blood (2008) 111 (9): 4523–4531.
https://doi.org/10.1182/blood-2007-10-120204
Published: 2008
Journal Articles

The Impact of Telomere Shortening in Dyskeratosis Congenita Cells on DNA Damage Response Pathways.

Travis Witt, BS, Aloysius Klingelhutz, PhD, Erik Westin, BS, Preeti Satyanarayana, MD, Peter M. Lansdorp, MD, PhD, Frederick D. Goldman, MD
Journal: Blood
Blood (2007) 110 (11): 4052.
https://doi.org/10.1182/blood.V110.11.4052.4052
Published: 2007
Journal Articles

Loss of Primitive Hematopoietic Cells in Patients with Dyskeratosis Congenita.

Geraldine Aubert, Frederick D. Goldman, Al J. Klingelhutz, Mark Hills, Sara R. Cooper, Wendy S. Hamilton, Annette Schlueter, Connie J. Eaves, Peter M. Lansdorp
Journal: Blood
Blood (2007) 110 (11): LB3.
https://doi.org/10.1182/blood.V110.11.LB3.lb3
Published: 2007
Journal Articles

The Role of Telomerase Activity and Telomere Length in Cellular Sensitivity to DNA Damaging Agents.

Greg T. Rice, BA, Michael A. Beasley, BS, Ike I. Akabogu, BS, Erik R. Westin, BS, Dale A. Winnike, BSN, Aloysius J. Klingelhutz, PHD, Frederick D. Goldman, MD
Journal: Blood
Blood (2006) 108 (11): 4192.
https://doi.org/10.1182/blood.V108.11.4192.4192
Published: 2006
Journal Articles

Development of hemophagocytic lymphohistiocytosis in triplets infected with HHV-8

Clinical Trials & Observations Brief Report
William J. Grossman, Mohammed Radhi, Dennis Schauer, Erick Gerday, Charles Grose, Frederick D. Goldman
Journal: Blood
Blood (2005) 106 (4): 1203–1206.
https://doi.org/10.1182/blood-2005-03-0950
Published: 2005
Journal Articles

Successful Unrelated Umbilical Cord Blood Transplantation for Shwachman-Diamond Syndrome.

Rajeev Vibhakar, MD, PhD, MPH, Stephen L. Rumelhart, PA-C, David J. Tatman, MS, Mohamed A. Radhi, MD, Frederick D. Goldman, MD, MS
Journal: Blood
Blood (2004) 104 (11): 5179.
https://doi.org/10.1182/blood.V104.11.5179.5179
Published: 2004
Journal Articles

Association of Telomere Length in Dyskeratosis Congenita Lymphocytes with Increased Sensitivity to Radiation and Anti-Mitotic Agents.

Michael A. Beasley, Vibha Singhal, MD, Aloysius J. Klingelhutz, PhD, Ike Akabogu, Frederick D. Goldman, MD
Journal: Blood
Blood (2004) 104 (11): 2833.
https://doi.org/10.1182/blood.V104.11.2833.2833
Published: 2004
Journal Articles

gp120-mediated induction of the MAPK cascade is dependent on the activation state of CD4 + lymphocytes

Sandrina Kinet, Fréderic Bernard, Cédric Mongellaz, Matthieu Perreau, Frederick D. Goldman, Naomi Taylor
Journal: Blood
Blood (2002) 100 (7): 2546–2553.
https://doi.org/10.1182/blood-2002-03-0819
Published: 2002