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Journal Articles

CXCR4 signaling controls dendritic cell location and activation at steady state and in inflammation

Carmen Gallego, Mathias Vétillard, Joseph Calmette, Mélanie Roriz, Viviana Marin-Esteban, Maximilien Evrard, Marie-Laure Aknin, Nicolas Pionnier, Manon Lefrançois, Françoise Mercier-Nomé, Yves Bertrand, Felipe Suarez, Jean Donadieu, Lai Guan Ng, Karl Balabanian, Françoise Bachelerie, Géraldine Schlecht-Louf

Blood (2021) 137 (20): 2770–2784.

https://doi.org/10.1182/blood.2020006675

Published: 2021

Includes: Supplemental data

Journal Articles

Family Studies of Whim Syndrome

David C. Dale, MD, Emily Dick, BA, Merideth L. Kelley, MSc, Vahagn Makaryan, MD, Jim Connelly, MD, Audrey Anna Bolyard, RN, BS

Blood (2019) 134 (Supplement_1): 215.

https://doi.org/10.1182/blood-2019-126234

Published: 2019

Journal Articles

Population Pharmacokinetic (PK)/Pharmacodynamic (PD) Modeling of Myelosuppression in Patients with Hematologic Malignancies for CPX-351 and Standard-of-Care 7+3 Therapy

Qi Wang, PhD, Sarah F. Cook, Scott A. Van Wart, Donald E. Mager, Stefan Faderl, MD

Blood (2018) 132 (Supplement 1): 4037.

https://doi.org/10.1182/blood-2018-99-111918

Published: 2018

Journal Articles

Spare the Spleen in ALPS: It Is Not an Expendable Vestigial Organ

V. Koneti Rao, MD FRCPA, Elaine Kulm, Susan Price, Sharon Webster, BSN, Les Folio, MD, Gulbu Uzel, MD, David T Teachey, MD

Blood (2018) 132 (Supplement 1): 2435.

https://doi.org/10.1182/blood-2018-99-117394

Published: 2018

Journal Articles

Determination of Phase 3 Dose for X4P-001 in Patients with WHIM Syndrome

David C Dale, MD, Frank Caleb Firkin, MD, Audrey Anna Bolyard, RN, BS, Merideth L. Kelley, MSc, Emily Dick, BA, Sonja Hartmann, PhD, Karen Brown, PhD, Tarek M Ebrahim, MD, Kenneth J Gorelick, MD, Sudha Parasuraman, MD

Blood (2018) 132 (Supplement 1): 1102.

https://doi.org/10.1182/blood-2018-99-119840

Published: 2018

Journal Articles

X4P-001: A Novel Molecularly-Targeted Oral Therapy for Whim Syndrome

David Dale, MD, Audrey Anna Bolyard, RN, BS, Emily Dick, BA, Merideth L. Kelley, MS, Vahagn Makaryan, MD, Ramsey Johnson, MSM, Lu Gan, MD PhD, Sudha Parasuraman, MD

Blood (2017) 130 (Supplement 1): 995.

https://doi.org/10.1182/blood.V130.Suppl_1.995.995

Published: 2017

Journal Articles

Clinical Correlates and Outcomes of Bone Marrow Failure and Myeloid Clonal Evolution in Patients with Germline GATA2 Haploinsufficiency

Candace Haddox, MD, Juliana Perez Botero, MD, Thanh Ho, Roshini Abraham, PhD, Shakila Khan, MD, Vilmarie Rodriguez, MD, Animesh Pardanani, MBBS PhD, Naseema Gangat, MBBS, William J. Hogan, MBBCh, Mark R. Litzow, MD, Matthew T Howard, MD, Mrinal M. Patnaik, MDMBBS

Blood (2017) 130 (Supplement 1): 2977.

https://doi.org/10.1182/blood.V130.Suppl_1.2977.2977

Published: 2017

Journal Articles

How I treat warts, hypogammaglobulinemia, infections, and myelokathexis syndrome

Raffaele Badolato, Jean Donadieu, the WHIM Research Group

Blood (2017) 130 (23): 2491–2498.

https://doi.org/10.1182/blood-2017-02-708552

Published: 2017

Journal Articles

Long-term outcome of hematopoietic stem cell transplantation for IL2RG/JAK3 SCID: a cohort report

Intan Juliana Abd Hamid, Mary A. Slatter, Fiona McKendrick, Mark S. Pearce, Andrew R. Gennery

Blood (2017) 129 (15): 2198–2201.

https://doi.org/10.1182/blood-2016-11-748616

Published: 2017

Includes: Supplemental data

Journal Articles

Clinical Spectrum of Germline Mutations with Predisposition to Myeloid Neoplasms- 2016 World Health Organization Classification Update

Thanh Ho, MD, Juliana Perez Botero, MD, William J Hogan, MBChB, Saad S Kenderian, MD, Naseema Gangat, MBBS, Ayalew Tefferi, MD, Roshini S Abraham, PhD, Phuong L. Nguyen, MD, Jennifer L Oliveira, MD, Rong He, MD, Dong Chen, MD PhD, David Viswanatha, MD, Vilmarie Rodriguez, MD, Shakila Khan, MD, Mrinal M Patnaik, MBBS

Blood (2016) 128 (22): 300.

https://doi.org/10.1182/blood.V128.22.300.300

Published: 2016

Journal Articles

Adenosine Deaminase 2 Deficiency As a Cause of Pure Red Cell Aplasia Mimicking Diamond Blackfan Anemia

Ghadir S. Sasa, MD, M. Tarek Elghetany, MD, Katie Bergstrom, Sarah Nicholas, MD, Ryan Himes, MD, Robert A. Krance, MD, Michael Hershfield, MD PhD, Joris van Montfrans, MD PhD, Alison Bertuch, MD PhD

Blood (2015) 126 (23): 3615.

https://doi.org/10.1182/blood.V126.23.3615.3615

Published: 2015

Journal Articles

Acute Lymphoblastic Leukemia in a Patient with Monomac Syndrome/GATA2 Haploinsufficiency

Ashley Koegel, MD, Venee N. Tubman, MD MMSc, Inga Hofmann, MD

Blood (2015) 126 (23): 3729.

https://doi.org/10.1182/blood.V126.23.3729.3729

Published: 2015

Journal Articles

Whim Syndrome Caused By a 5 Base Pair Deletion in the C-Terminus of Chemokine Receptor CXCR4

Qian Liu, MD, Lizbeeth Lopez, Catherina Pan, Daniel Velez, John S. Corns, Donald T. Ellenburg, Harry L. Malech, MD, Philip M. Murphy, MD, David H. McDermott, MD

Blood (2015) 126 (23): 1003.

https://doi.org/10.1182/blood.V126.23.1003.1003

Published: 2015

Journal Articles

CXCR4 Gene Dosage Is Critical for HSC Engraftment

David H. McDermott, MD, Paejonette Jacobs, PhD, Qian Liu, MD, Jiliang Gao, PhD, Philip M. Murphy, MD

Blood (2015) 126 (23): 3066.

https://doi.org/10.1182/blood.V126.23.3066.3066

Published: 2015

Journal Articles

Lentiviral Hematopoietic Stem Cell Gene Therapy for Older Patients with X-Linked Severe Combined Immunodeficiency

Suk See De Ravin, MDPhD, Xiaolin Wu, Narda Theobald, Janet S Lee, John Gray, PhD, Imelda Celine Hanson, MD, Luigi Notarangelo, MDPhD, Brian P. Sorrentino, MD, Harry L. Malech, MD

Blood (2015) 126 (23): 261.

https://doi.org/10.1182/blood.V126.23.261.261

Published: 2015

Journal Articles

Allogeneic Stem Cell Transplantation in a Pediatric Patient with Whim Syndrome

Saleh Bhar, MD, Khaled Yassine, MD, Caridad Martinez, MD, Ghadir S. Sasa, MD, Swati Naik, MD, Donald Mahoney, Jr., MD, Carl Allen, MD PhD, Nabil Ahmed, MD, Meenakshi G. Hegde, MD, Helen E. Heslop, MD, Stephen Gottschalk, MD, Robert A. Krance, MD, Kathryn S. Leung

Blood (2015) 126 (23): 5528.

https://doi.org/10.1182/blood.V126.23.5528.5528

Published: 2015

Journal Articles

How I treat Waldenström macroglobulinemia

Steven P. Treon

Blood (2015) 126 (6): 721–732.

https://doi.org/10.1182/blood-2015-01-553974

Published: 2015

Journal Articles

Specific Impaired CD56+ NK Cell Development in GATA2 Deficiency As Revealed By Ex Vivo NK Expansion

Moonjung Jung, MD, Jan K. Davidson-Moncada, MD PhD, Chuanfeng Wu, PhD, Samson Koelle, BA, Thomas Winkler, MD, Danielle M. Townsley, MD, Amy Hsu, BS, Keyvan Keyvanfar, BS, Steven M. Holland, MD, Dennis D. Hickstein, MD, Richard W. Childs, MD, Cynthia E. Dunbar, MD

Blood (2014) 124 (21): 1419.

https://doi.org/10.1182/blood.V124.21.1419.1419

Published: 2014

Journal Articles

Spectrum of Phenotypic Manifestations of a Novel c1339A>C, pS447R Germline GATA2 Mutation

Samith T. Kochuparambil, MBBS, Amie E Jackson, MD, Mark R. Litzow, MD, Shakila Khan, MD, Vilmarie Rodriguez, MD, William J. Hogan, MBChB, Roshini Abraham, PhD, Mrinal M. Patnaik, MBBS

Blood (2014) 124 (21): 5614.

https://doi.org/10.1182/blood.V124.21.5614.5614

Published: 2014

Journal Articles

Defect in B Cell Production Driven By GATA2 Mutation Results in Their Absolute Reduction and Mature Phenotype in Pediatric Patients

Michaela Novakova, Ales Janda, Marcin W Wlodarski, Martina Sukova, Vit Campr, Marketa Kubricanova Zaliova, Eva Fronkova, Elena Vodickova, Ondrej Zapletal, Jan Blatny, Katerina Lejhancova, Dagmar Pospisilova, Zdena Cerna, Tomas Kuhn, Zuzana Zemanova, Jan Trka, Ondrej Hrusak, Jan Stary, Ester Mejstrikova

Blood (2014) 124 (21): 2746.

https://doi.org/10.1182/blood.V124.21.2746.2746

Published: 2014

NARROW

CXCR4 signaling controls dendritic cell location and activation at steady state and in inflammation

Family Studies of Whim Syndrome

Population Pharmacokinetic (PK)/Pharmacodynamic (PD) Modeling of Myelosuppression in Patients with Hematologic Malignancies for CPX-351 and Standard-of-Care 7+3 Therapy

Spare the Spleen in ALPS: It Is Not an Expendable Vestigial Organ

Determination of Phase 3 Dose for X4P-001 in Patients with WHIM Syndrome

X4P-001: A Novel Molecularly-Targeted Oral Therapy for Whim Syndrome

Clinical Correlates and Outcomes of Bone Marrow Failure and Myeloid Clonal Evolution in Patients with Germline GATA2 Haploinsufficiency

How I treat warts, hypogammaglobulinemia, infections, and myelokathexis syndrome

Long-term outcome of hematopoietic stem cell transplantation for IL2RG/JAK3 SCID: a cohort report

Clinical Spectrum of Germline Mutations with Predisposition to Myeloid Neoplasms- 2016 World Health Organization Classification Update

Adenosine Deaminase 2 Deficiency As a Cause of Pure Red Cell Aplasia Mimicking Diamond Blackfan Anemia

Acute Lymphoblastic Leukemia in a Patient with Monomac Syndrome/GATA2 Haploinsufficiency

Whim Syndrome Caused By a 5 Base Pair Deletion in the C-Terminus of Chemokine Receptor CXCR4

CXCR4 Gene Dosage Is Critical for HSC Engraftment

Lentiviral Hematopoietic Stem Cell Gene Therapy for Older Patients with X-Linked Severe Combined Immunodeficiency

Allogeneic Stem Cell Transplantation in a Pediatric Patient with Whim Syndrome

How I treat Waldenström macroglobulinemia

Specific Impaired CD56+ NK Cell Development in GATA2 Deficiency As Revealed By Ex Vivo NK Expansion

Spectrum of Phenotypic Manifestations of a Novel c1339A>C, pS447R Germline GATA2 Mutation

Defect in B Cell Production Driven By GATA2 Mutation Results in Their Absolute Reduction and Mature Phenotype in Pediatric Patients

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American Society of Hematology

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American Society of Hematology

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