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Molecular Therapy. Methods & Clinical Development
|
September 6, 2021
Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency
Sara Fañanas-Baquero, Oscar Quintana-Bustamante, Daniel P Dever, et al.
The Journal of Experimental Medicine
|
September 29, 2023
Epitope-engineered human hematopoietic stem cells are shielded from CD123-targeted immunotherapy
Romina Marone, Emmanuelle Landmann, Anna Devaux, et al.
Nature Medicine
|
July 22, 2025
Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial
Rajni Agarwal, Alice Bertaina, Charmaine Soco, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
February 2, 2013
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases
Jacques P Tremblay, Xiao Xiao, Annemieke Aartsma-Rus, et al.
JAMA
|
August 21, 2014
Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States
Antonia Kwan, Roshini S Abraham, Robert Currier, et al.
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Search research articles
Search
Showing results (41-50 of 45) with videos related to
Sort By:
Page
of 5
You have reached the last page of results.
This site can display upto 45 results.
Molecular Therapy. Methods & Clinical Development
|
September 6, 2021
Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency
Sara Fañanas-Baquero, Oscar Quintana-Bustamante, Daniel P Dever, et al.
The Journal of Experimental Medicine
|
September 29, 2023
Epitope-engineered human hematopoietic stem cells are shielded from CD123-targeted immunotherapy
Romina Marone, Emmanuelle Landmann, Anna Devaux, et al.
Nature Medicine
|
July 22, 2025
Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial
Rajni Agarwal, Alice Bertaina, Charmaine Soco, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
February 2, 2013
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases
Jacques P Tremblay, Xiao Xiao, Annemieke Aartsma-Rus, et al.
JAMA
|
August 21, 2014
Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States
Antonia Kwan, Roshini S Abraham, Robert Currier, et al.
Page
of 5