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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

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

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

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

Journal Articles

DNA Methyltransferase Inhibitors Promote Homologous Recombination Deficiency through Induction of Immune Signaling, Sensitizing Acute Myeloid Leukemia Cells to PARP Inhibitors

Aksinija A Kogan, BS, MS, Lena J Mclaughlin, BS, Maria R. Baer, MD, Stephen Baylin, MD, Michael Topper, BS, Feyruz V. Rassool, BSc, PhD

Blood (2019) 134 (Supplement_1): 3763.

https://doi.org/10.1182/blood-2019-129535

Published: 2019

Journal Articles

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

Agnieszka Czechowicz, MD PhD, Maria Grazia Roncarolo, MD, Brian C Beard, PhD, Ken Law, PhD, Eileen Nicoletti, MD, Paula Río, PhD, Juan A. Bueren, PhD, Jonathan D Schwartz, MD, Sandeep Soni, MBBS

Blood (2019) 134 (Supplement_1): 3350.

https://doi.org/10.1182/blood-2019-127352

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

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

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

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

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

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

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

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.

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

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

Phase II Study of Eltrombopag in Subjects with Fanconi Anemia

DNA Methyltransferase Inhibitors Promote Homologous Recombination Deficiency through Induction of Immune Signaling, Sensitizing Acute Myeloid Leukemia Cells to PARP Inhibitors

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

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

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

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

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

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

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

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American Society of Hematology

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American Society of Hematology

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