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Journal Articles

Analysis of disease model iPSCs derived from patients with a novel Fanconi anemia–like IBMFS ADH5/ALDH2 deficiency

Anfeng Mu, Asuka Hira, Akira Niwa, Mitsujiro Osawa, Kenichi Yoshida, Minako Mori, Yusuke Okamoto, Kazuko Inoue, Keita Kondo, Masato T. Kanemaki, Tomonari Matsuda, Etsuro Ito, Seiji Kojima, Tatsutoshi Nakahata, Seishi Ogawa, Keigo Tanaka, Keitaro Matsuo, Megumu K. Saito, Minoru Takata

Blood (2021) 137 (15): 2021–2032.

https://doi.org/10.1182/blood.2020009111

Published: 2021

Includes: Supplemental data

Journal Articles

Association of clinical severity with FANCB variant type in Fanconi anemia

Moonjung Jung, Ramanagouda Ramanagoudr-Bhojappa, Sylvie van Twest, Rasim Ozgur Rosti, Vincent Murphy, Winnie Tan, Frank X. Donovan, Francis P. Lach, Danielle C. Kimble, Caroline S. Jiang, Roger Vaughan, Parinda A. Mehta, Filomena Pierri, Carlo Dufour, Arleen D. Auerbach, Andrew J. Deans, Agata Smogorzewska, Settara C. Chandrasekharappa

Blood (2020) 135 (18): 1588–1602.

https://doi.org/10.1182/blood.2019003249

Published: 2020

Includes: Supplemental data

Journal Articles

ILF2 Antisense Oligonucleotide Therapy and a CRISPR/Cas9-Based Screening for DNA Repair Effectors Identify Synthetic Lethal Approaches Enhancing Myeloma Cells Sensitivity to DNA Damage

Natthakan Thongon, PhD, Christopher Jackson, BS, Pamela Lockyer, MS, Jintan Liu, Andrea Santoni, PhD, Ashley Rose, BS, Guillermo Garcia-Manero, MD, Simona Colla, PhD

Blood (2019) 134 (Supplement_1): 685.

https://doi.org/10.1182/blood-2019-129286

Published: 2019

Journal Articles

Mapping the Evolution of the Mutational Landscape of Acute Myeloid Leukemia from Diagnosis to Post-Transplantation Relapse and Its Interplay with Immune Evasion Mechanisms

Elisa Sala, MD, Francesca Biavasco, MD, Gabriele Bucci, PhD, Cristina Toffalori, PhD, Donatella Biancolini, PhD, Elena Antonini, MSc, Matteo Giovanni Carrabba, MD, Bernhard Gentner, MD, Cristina Tresoldi, MSc, Massimo Bernardi, MD, Dejan Lazarevic, MD, Giovanni Tonon, MD, Fabio Ciceri, MD, Luca Vago, MD PhD

Blood (2019) 134 (Supplement_1): 511.

https://doi.org/10.1182/blood-2019-124725

Published: 2019

Journal Articles

FA Gene Carrier Status Predisposes to Myeloid Neoplasms and Bone Marrow Failure in Adults

Jibran Durrani, Hassan Awada, MD, Wenyi Shen, MD, Cassandra M Kerr, MS, Vera Adema, PhD, Sunisa Kongkiatkamon, Ashwin Kishtagari, MBBS, Samuel Li, Yasunobu Nagata, MD PhD, Sanghee Hong, MD, Aziz Nazha, MD, Bhumika J. Patel, MD, Yogenthiran Saunthararajah, MD, Hetty E. Carraway, MD MBA, Valeria Visconte, PhD, Seth J. Corey, MD, Mikkael A. Sekeres, MD MS, Thomas Laframboise, PhD, Jaroslaw P. Maciejewski, MD PhD

Blood (2019) 134 (Supplement_1): 452.

https://doi.org/10.1182/blood-2019-130142

Published: 2019

Journal Articles

No Evidence for Hematopoietic Stem Cell Self-Renewal in-Vivo Following Inflammatory Challenge

Blood (2019) 134 (Supplement_1): 456.

https://doi.org/10.1182/blood-2019-125744

Published: 2019

Journal Articles

A New Step in Understanding of Fanconi Patients Peripheral Stem Cell Harvesting, a Bridge to Gene Therapy

Jean-Sebastien Diana, MD, Sandra Manceau, PhD, Thierry Leblanc, MD, Chloé Couzin, MD, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Matthieux Bendavid, MD, Laure Joseph, MD, Marianne Delville, MD, Stephane Blanche, MD, Jean Soulier, MD PhD, Marina Cavazzana, MD PhD, Francois Lefrere, MD

Blood (2019) 134 (Supplement_1): 1970.

https://doi.org/10.1182/blood-2019-128051

Published: 2019

Journal Articles

Potential Influence of Hematological or Immunological Hereditary Predisposition Genes on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Hematological Malignancies

Zhihui Li, Qinlong Zheng, Yongqiang Zhao, Yanzhi Song, Fan Yang, Min Jiang, Chunrong Tong, Tong Wu, MD

Blood (2019) 134 (Supplement_1): 2037.

https://doi.org/10.1182/blood-2019-126339

Published: 2019

Journal Articles

Genetics of Monosomy 7 and Del(7q) in MDS Informs Potential Therapeutic Targets

Cassandra M Kerr, MS, Vera Adema, PhD, Wencke Walter, PhD, Stephan Hutter, PhD, Christina A. Snider, BA, Yasunobu Nagata, MD PhD, Manja Meggendorfer, PhD, Claudia Haferlach, MD, Valeria Visconte, PhD, Mikkael A. Sekeres, MD MS, Thomas Laframboise, PhD, Torsten Haferlach, MD, Jaroslaw P. Maciejewski, MD PhD

Blood (2019) 134 (Supplement_1): 1703.

https://doi.org/10.1182/blood-2019-126866

Published: 2019

Journal Articles

UAF1 DNA Binding Activity Is Critical for RAD51-Mediated Homologous DNA Pairing

Fengshan Liang, PhD, Adam S Miller, PhD, Carolilne Tang, Patrick Sung, PhD, Gary M. Kupfer, MD

Blood (2019) 134 (Supplement_1): 2497.

https://doi.org/10.1182/blood-2019-130435

Published: 2019

Journal Articles

Efficacy and Safety of a FLAG-Sequential Regimen Followed By Hematopoietic Stem Cell Transplantation for Myelodysplasia or Acute Leukemia in Fanconi Anemia: A Franco-Brazilian Report

Pierre-Edouard Debureaux, Flore Sicre de Fontbrune, Carmem M. S. Bonfim, MD, Jean-Hugues Dalle, MD PhD, Nimrod Buchbinder, MD, Yves Bertrand, MD, Cecile Renard, MD, Edouard Forcade, MD, Juliana Fernandes, MD, Alexis Talbot, MD, Ricardo Pasquini, MD, Jean Soulier, MD PhD, Thierry Leblanc, MD, Gerard Socie, MD PhD, Regis Peffault de Latour, MD PhD

Blood (2019) 134 (Supplement_1): 2498.

https://doi.org/10.1182/blood-2019-122007

Published: 2019

Journal Articles

Predictors of Disease Progression and Survival in Patients with Myelodysplastic Syndrome Secondary to Inherited Bone Marrow Failure Syndromes

Blood (2019) 134 (Supplement_1): 2507.

https://doi.org/10.1182/blood-2019-130129

Published: 2019

Journal Articles

Cellular and Molecular Architecture of Hematopoietic Stem Cells and Progenitors in Genetic Models of Bone Marrow Failure

Stephanie C. Heidemann, MD, Brian Bursic, Sasan Zandi, MD PhD, Hongbing Li, PhD, Robert J Klaassen, MD FRCPC, Sharon Abish, MD, Meera Rayar, Vicky R. Breakey, BSc,MDFRCPC, Houtan Moshiri, PhD, Santhosh Dhanraj, PhD, Richard de Borja, PhD, Adam Shlien, PhD, John E. Dick, PhD, Yigal Dror, MDFRCPC, Sagi Abelson, PhD

Blood (2019) 134 (Supplement_1): 1223.

https://doi.org/10.1182/blood-2019-130534

Published: 2019

Journal Articles

NHEJ-Mediated Gene Editing, a Versatile Approach to Correct a Variety of Fanconi Anemia Genes in HSCs

Francisco J Roman-Rodriguez, PhD, Laura Ugalde, Lara A. Alvarez, Begoña Díez-Cabezas, PhD, Jordi Surrallés, PhD, Maria Jose Ramirez, PhD, Massimo Bogliolo, PhD, Helmut Hanenberg, MD PhD, Julian Sevilla, MD PhD, Sandra Rodriguez-Perales, PhD, Raul Torres-Ruiz, PhD, Juan A. Bueren, PhD, Paula Rio, PhD

Blood (2019) 134 (Supplement_1): 4639.

https://doi.org/10.1182/blood-2019-129946

Published: 2019

Journal Articles

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Blood (2019) 134 (Supplement_1): 4627.

https://doi.org/10.1182/blood-2019-129686

Published: 2019

Journal Articles

Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency

Rajni Agarwal, MD, Christopher C. Dvorak, MD, Hye-Sook Kwon, PhD, Janel R Long-Boyle, PharmD, PhD, Susan S Prohaska, PhD MS, Janice W Brown, MD, Anne Le, Alyssa Guttman-Klein, Irving L Weissman, MD, Morton J Cowan, MD, Aaron C. Logan, MD PhD, Kenneth I. Weinberg, MD, Robertson Parkman, MD, Maria-Grazia Roncarolo, MD, Judith A Shizuru, MD PhD

Blood (2019) 134 (Supplement_1): 800.

https://doi.org/10.1182/blood-2019-126239

Published: 2019

Journal Articles

Cells Deficient in the Shwachman-Diamond Syndrome Protein SBDS or the Diamond-Blackfan Anemia Protein RPS19 Have Impaired Homologous Recombination

Elif Asik, PhD, Nimrat Chatterjee, PhD, Alison A. Bertuch, MD PhD

Blood (2019) 134 (Supplement_1): 104.

https://doi.org/10.1182/blood-2019-128953

Published: 2019

Journal Articles

Pharmacologic Activation of Aldehyde Metabolism to Protect Hematopoietic Stem Cells (HSC) in Murine Models of Fanconi Anemia (FA)

Jennifer Tsai, MD, Kelsey R. Logas, MS, Lauren D. Van Wassenhove, PhD, Beruh Dejene, BS, Che-Hong Chen, PhD, Daria Mochly-Rosen, PhD, Kenneth I. Weinberg, MD

Blood (2019) 134 (Supplement_1): 105.

https://doi.org/10.1182/blood-2019-124439

Published: 2019

Journal Articles

A Novel Source of Endogenous DNA Damage That Requires Repair By the Fanconi Anemia Pathway

Moonjung Jung, MD, Sunandini Sridhar, PhD, Audrey Goldfarb, BA, Ryan White, PhD, Danielle Keahi, BA, Raymond Noonan, BA, Tom Wiley, BS, Francis P. Lach, BS, Agata Smogorzewska, MD PhD

Blood (2019) 134 (Supplement_1): 106.

https://doi.org/10.1182/blood-2019-127686

Published: 2019

Journal Articles

Replication Stress Response and CDKN1A Engagement Constrain Fetal Hematopoietic Stem Cell Pool Size in Fanconi Anemia

Makiko Mochizuki-Kashio, PhD, Young Me Yoon, Theresa N Menna, BS, Markus Grompe, MD, Peter Kurre, MD

Blood (2019) 134 (Supplement_1): 107.

https://doi.org/10.1182/blood-2019-125669

Published: 2019

NARROW

Analysis of disease model iPSCs derived from patients with a novel Fanconi anemia–like IBMFS ADH5/ALDH2 deficiency

Association of clinical severity with FANCB variant type in Fanconi anemia

ILF2 Antisense Oligonucleotide Therapy and a CRISPR/Cas9-Based Screening for DNA Repair Effectors Identify Synthetic Lethal Approaches Enhancing Myeloma Cells Sensitivity to DNA Damage

Mapping the Evolution of the Mutational Landscape of Acute Myeloid Leukemia from Diagnosis to Post-Transplantation Relapse and Its Interplay with Immune Evasion Mechanisms

FA Gene Carrier Status Predisposes to Myeloid Neoplasms and Bone Marrow Failure in Adults

No Evidence for Hematopoietic Stem Cell Self-Renewal in-Vivo Following Inflammatory Challenge

A New Step in Understanding of Fanconi Patients Peripheral Stem Cell Harvesting, a Bridge to Gene Therapy

Potential Influence of Hematological or Immunological Hereditary Predisposition Genes on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Hematological Malignancies

Genetics of Monosomy 7 and Del(7q) in MDS Informs Potential Therapeutic Targets

UAF1 DNA Binding Activity Is Critical for RAD51-Mediated Homologous DNA Pairing

Efficacy and Safety of a FLAG-Sequential Regimen Followed By Hematopoietic Stem Cell Transplantation for Myelodysplasia or Acute Leukemia in Fanconi Anemia: A Franco-Brazilian Report

Predictors of Disease Progression and Survival in Patients with Myelodysplastic Syndrome Secondary to Inherited Bone Marrow Failure Syndromes

Cellular and Molecular Architecture of Hematopoietic Stem Cells and Progenitors in Genetic Models of Bone Marrow Failure

NHEJ-Mediated Gene Editing, a Versatile Approach to Correct a Variety of Fanconi Anemia Genes in HSCs

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency

Cells Deficient in the Shwachman-Diamond Syndrome Protein SBDS or the Diamond-Blackfan Anemia Protein RPS19 Have Impaired Homologous Recombination

Pharmacologic Activation of Aldehyde Metabolism to Protect Hematopoietic Stem Cells (HSC) in Murine Models of Fanconi Anemia (FA)

A Novel Source of Endogenous DNA Damage That Requires Repair By the Fanconi Anemia Pathway

Replication Stress Response and CDKN1A Engagement Constrain Fetal Hematopoietic Stem Cell Pool Size in Fanconi Anemia

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American Society of Hematology

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ASH Publications

American Society of Hematology

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