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Science Translational Medicine
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April 22, 2016
Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency
Suk See De Ravin, Xiaolin Wu, Susan Moir, et al.
Nature
|
April 22, 2025
Custom CRISPR-Cas9 PAM variants via scalable engineering and machine learning
Rachel A Silverstein, Nahye Kim, Ann-Sophie Kroell, et al.
Blood Advances
|
December 7, 2020
NADPH oxidase correction by mRNA transfection of apheresis granulocytes in chronic granulomatous disease
Suk See De Ravin, Julie Brault, Ronald J Meis, et al.
The New England Journal of Medicine
|
October 15, 2025
Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency
Claire Booth, Katelyn Masiuk, Konstantinos Vazouras, et al.
Science Translational Medicine
|
February 7, 2024
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency
Maria Carmina Castiello, Chiara Brandas, Samuele Ferrari, et al.
The New England Journal of Medicine
|
April 18, 2019
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1
Ewelina Mamcarz, Sheng Zhou, Timothy Lockey, et al.
Blood
|
May 11, 2021
Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency
Bryanna Reinhardt, Omar Habib, Kit L Shaw, et al.
The New England Journal of Medicine
|
December 8, 2025
Prime Editing for p47<sup>phox</sup>-Deficient Chronic Granulomatous Disease
Jennifer L Gori, Elie Haddad, Haydar Frangoul, et al.
Blood
|
January 29, 2021
Immunodeficiency and bone marrow failure with mosaic and germline TLR8 gain of function
Jahnavi Aluri, Alicia Bach, Saara Kaviany, et al.
Nature Medicine
|
January 29, 2020
Lentiviral gene therapy for X-linked chronic granulomatous disease
Donald B Kohn, Claire Booth, Elizabeth M Kang, et al.
Page
of 7
Search research articles
Search
Showing results (51-60 of 66) with videos related to
Sort By:
Page
of 7
Science Translational Medicine
|
April 22, 2016
Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency
Suk See De Ravin, Xiaolin Wu, Susan Moir, et al.
Nature
|
April 22, 2025
Custom CRISPR-Cas9 PAM variants via scalable engineering and machine learning
Rachel A Silverstein, Nahye Kim, Ann-Sophie Kroell, et al.
Blood Advances
|
December 7, 2020
NADPH oxidase correction by mRNA transfection of apheresis granulocytes in chronic granulomatous disease
Suk See De Ravin, Julie Brault, Ronald J Meis, et al.
The New England Journal of Medicine
|
October 15, 2025
Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency
Claire Booth, Katelyn Masiuk, Konstantinos Vazouras, et al.
Science Translational Medicine
|
February 7, 2024
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency
Maria Carmina Castiello, Chiara Brandas, Samuele Ferrari, et al.
The New England Journal of Medicine
|
April 18, 2019
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1
Ewelina Mamcarz, Sheng Zhou, Timothy Lockey, et al.
Blood
|
May 11, 2021
Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency
Bryanna Reinhardt, Omar Habib, Kit L Shaw, et al.
The New England Journal of Medicine
|
December 8, 2025
Prime Editing for p47<sup>phox</sup>-Deficient Chronic Granulomatous Disease
Jennifer L Gori, Elie Haddad, Haydar Frangoul, et al.
Blood
|
January 29, 2021
Immunodeficiency and bone marrow failure with mosaic and germline TLR8 gain of function
Jahnavi Aluri, Alicia Bach, Saara Kaviany, et al.
Nature Medicine
|
January 29, 2020
Lentiviral gene therapy for X-linked chronic granulomatous disease
Donald B Kohn, Claire Booth, Elizabeth M Kang, et al.
Page
of 7