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Journal Articles

Gene therapy for hemophilia

Amit C. Nathwani
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2019) 2019 (1): 1–8.
https://doi.org/10.1182/hematology.2019000007
Published: 2019
Journal Articles

Treatment of primary immunodeficiency with allogeneic transplant and gene therapy

Sung-Yun Pai
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2019) 2019 (1): 457–465.
https://doi.org/10.1182/hematology.2019000052
Published: 2019
Journal Articles

Optimal disease management and health monitoring in adults with sickle cell disease

Jo Howard, Swee Lay Thein
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2019) 2019 (1): 505–512.
https://doi.org/10.1182/hematology.2019000055
Published: 2019
Journal Articles

Beyond transfusion therapy: new therapies in thalassemia including drugs, alternate donor transplant, and gene therapy

John Porter
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2018) 2018 (1): 361–370.
https://doi.org/10.1182/asheducation-2018.1.361
Published: 2018
Journal Articles

New therapeutic targets in transfusion-dependent and -independent thalassemia

M. Domenica Cappellini, Irene Motta
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2017) 2017 (1): 278–283.
https://doi.org/10.1182/asheducation-2017.1.278
Published: 2017
Journal Articles

Hemophilia gene therapy comes of age

Lindsey A. George
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2017) 2017 (1): 587–594.
https://doi.org/10.1182/asheducation-2017.1.587
Published: 2017
Journal Articles

Novel alternate hemostatic agents for patients with inhibitors: beyond bypass therapy

Margaret V. Ragni
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2017) 2017 (1): 605–609.
https://doi.org/10.1182/asheducation-2017.1.605
Published: 2017
Journal Articles

Emerging genetic and pharmacologic therapies for controlling hemostasis: beyond recombinant clotting factors

Paul E. Monahan
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2015) 2015 (1): 33–40.
https://doi.org/10.1182/asheducation-2015.1.33
Published: 2015
Journal Articles

Replacing bad (F)actors: hemophilia

Christopher B. Doering, H. Trent Spencer
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2014) 2014 (1): 461–467.
https://doi.org/10.1182/asheducation-2014.1.461
Published: 2014
Journal Articles

Eliminating SCID row: new approaches to SCID

Donald B. Kohn
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2014) 2014 (1): 475–480.
https://doi.org/10.1182/asheducation-2014.1.475
Published: 2014
Journal Articles

Immunotherapeutic strategies including transplantation: eradication of disease

John G. Gribben, John C. Riches
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2013) 2013 (1): 151–157.
https://doi.org/10.1182/asheducation-2013.1.151
Published: 2013
Journal Articles

The gene therapy journey for hemophilia: are we there yet?

Katherine A. High
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2012) 2012 (1): 375–381.
https://doi.org/10.1182/asheducation.V2012.1.375.3797885
Published: 2012
Journal Articles

Advances in stem cell transplantation and gene therapy in the β-hemoglobinopathies

Emmanuel Payen, Philippe Leboulch
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2012) 2012 (1): 276–283.
https://doi.org/10.1182/asheducation.V2012.1.276.3807841
Published: 2012
Journal Articles

Alternative Strategies for Gene Therapy of Hemophilia

Robert R. Montgomery, Qizhen Shi
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2010) 2010 (1): 197–202.
https://doi.org/10.1182/asheducation-2010.1.197
Published: 2010
Journal Articles

Genetic Therapy for Beta-Thalassemia: From the Bench to the Bedside

Paritha Arumugam, Punam Malik
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2010) 2010 (1): 445–450.
https://doi.org/10.1182/asheducation-2010.1.445
Published: 2010
Journal Articles

Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders

Derek A. Persons
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2009) 2009 (1): 690–697.
https://doi.org/10.1182/asheducation-2009.1.690
Published: 2009
Journal Articles

Overview of gene therapy clinical progress including cancer treatment with gene-modified T cells

Malcolm K. Brenner, Fatma V. Okur
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2009) 2009 (1): 675–681.
https://doi.org/10.1182/asheducation-2009.1.675
Published: 2009
Journal Articles

Ten years of gene therapy for primary immune deficiencies

Alessandro Aiuti, Maria Grazia Roncarolo
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2009) 2009 (1): 682–689.
https://doi.org/10.1182/asheducation-2009.1.682
Published: 2009
Journal Articles

Gene Replacement Therapy for Sickle Cell Disease and Other Blood Disorders

Tim M. Townes
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2008) 2008 (1): 193–196.
https://doi.org/10.1182/asheducation-2008.1.193
Published: 2008
Journal Articles

The Yin and Yang of Stem Cell Gene Therapy: Insights into Hematopoiesis, Leukemogenesis, and Gene Therapy Safety

Cynthia E. Dunbar
Journal: Hematology
Hematology Am Soc Hematol Educ Program (2007) 2007 (1): 460–465.
https://doi.org/10.1182/asheducation-2007.1.460
Published: 2007