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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

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

Journal Articles

The Path to Cure: Using Haploidentical (haplo) Donors and High-Dose Post-Transplant Cyclophosphamide (PTCy) for Treatment-Naïve and Refractory Severe Aplastic Anemia (SAA)

Amy E. DeZern, MD, Marianna Zahurak, MS, Kenneth R. Cooke, MD, David Margolis, MD, Richard J. Jones, MD, Robert A. Brodsky, MD

Blood (2019) 134 (Supplement_1): 147.

https://doi.org/10.1182/blood-2019-121900

Published: 2019

Journal Articles

The Impact of Identifying the Syndromic and Genetic Diagnoses on Hematopoietic Stem Cell Transplantation Outcome in Patients with Inherited Bone Marrow Failure Syndromes

Blood (2019) 134 (Supplement_1): 5015.

https://doi.org/10.1182/blood-2019-121743

Published: 2019

Journal Articles

Myeloid Tumor-Related Somatic Mutations in 293 Children and Adults with Acquired Aplastic Anemia in Chinese

Hailong He, MD, Hua Yin, Xiaoqing Liu, Lang Cheng, Guangsheng He, Jianyong Li, MD

Blood (2019) 134 (Supplement_1): 5024.

https://doi.org/10.1182/blood-2019-129813

Published: 2019

Journal Articles

Germinal Predisposition in Myelodysplastic Syndromes in Young Adults without a Preexisting Disorder or Organ Dysfunction: Identification of Deleterious Variants in Microsatellite Instability Genes

Blood (2019) 134 (Supplement_1): 4226.

https://doi.org/10.1182/blood-2019-127204

Published: 2019

Journal Articles

Fanconi Anemia: A Paradigm for Understanding DNA Repair During Replication.

Agata Smogorzewska, MD PhD

Blood (2019) 134 (Supplement_1): SCI-32.

https://doi.org/10.1182/blood-2019-121229

Published: 2019

Journal Articles

Survival Outcomes Following Allogeneic Stem Cell Transplantation for Inherited Bone Marrow Failure and Myeloid Germline Predisposition Syndromes

Kalyan Nadiminti, Abhishek A Mangaonkar, MBBS, Kimberly J. Langer, D.N.P., C.N.P., Shakila P Khan, MD, Vilmarie Rodriguez, MD, Naseema Gangat, MBBS, Michelle A Elliott, MD, Hassan B. Alkhateeb, MD, Mithun Vinod Shah, MD PhD, Mark Litzow, MD, William J Hogan, MB, BCh, Saad S. Kenderian, MD, Mrinal M Patnaik, MDMBBS

Blood (2019) 134 (Supplement_1): 3300.

https://doi.org/10.1182/blood-2019-121742

Published: 2019

Journal Articles

Familial Clustering of Hematological Malignancies: Harbingers of Wider Germline Cancer Susceptibility

Anna L Brown, PhD, Milena Babic, BMPharBio (Hons), Andreas Schreiber, Jinghua Feng, PhD, Julia Dobbins, BSc (Hons), Peer Arts, PhD, Paul Wang, PhD, Kerry Phillips, Devendra K Hiwase, MBBS, MD FRACP, FRCPA, PhD, Parvathy Venugopal, PhD MSc, BSc, Susan Branford, PhD FFSc (RCPA), Nicola Poplawski, MD, Christopher N Hahn, PhD, Hamish S Scott, PhD FFSc (RCPA)

Blood (2019) 134 (Supplement_1): 3794.

https://doi.org/10.1182/blood-2019-131642

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

The Fanconi Anemia-Associated Protein NIPA Is Essential for the Nuclear Abundance of FANCD2

Blood (2019) 134 (Supplement_1): 3741.

https://doi.org/10.1182/blood-2019-125973

Published: 2019

Journal Articles

Phase II Study of Eltrombopag in Subjects with Fanconi Anemia

Daisuke Araki, MD, Ma Evette Barranta, Fariba Chinian, MS, Julie Erb-Alvarez, MPH, Thomas Winkler, MD, Neelam Giri, MD, Andre Larochelle, MD PhD

Blood (2019) 134 (Supplement_1): 3753.

https://doi.org/10.1182/blood-2019-123151

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

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

The Path to Cure: Using Haploidentical (haplo) Donors and High-Dose Post-Transplant Cyclophosphamide (PTCy) for Treatment-Naïve and Refractory Severe Aplastic Anemia (SAA)

The Impact of Identifying the Syndromic and Genetic Diagnoses on Hematopoietic Stem Cell Transplantation Outcome in Patients with Inherited Bone Marrow Failure Syndromes

Myeloid Tumor-Related Somatic Mutations in 293 Children and Adults with Acquired Aplastic Anemia in Chinese

Germinal Predisposition in Myelodysplastic Syndromes in Young Adults without a Preexisting Disorder or Organ Dysfunction: Identification of Deleterious Variants in Microsatellite Instability Genes

Fanconi Anemia: A Paradigm for Understanding DNA Repair During Replication.

Survival Outcomes Following Allogeneic Stem Cell Transplantation for Inherited Bone Marrow Failure and Myeloid Germline Predisposition Syndromes

Familial Clustering of Hematological Malignancies: Harbingers of Wider Germline Cancer Susceptibility

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

The Fanconi Anemia-Associated Protein NIPA Is Essential for the Nuclear Abundance of FANCD2

Phase II Study of Eltrombopag in Subjects with Fanconi Anemia

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American Society of Hematology

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ASH Publications

American Society of Hematology

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