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Human Gene Therapy Methods
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August 22, 2014
Copackaging of multiple adeno-associated viral vectors in a single production step
Phillip A Doerfler, Barry J Byrne, Nathalie Clément
Clinical Immunology (Orlando, Fla.)
|
April 6, 2015
BAFF blockade prevents anti-drug antibody formation in a mouse model of Pompe disease
Phillip A Doerfler, Sushrusha Nayak, Roland W Herzog, et al.
Molecular Therapy. Methods & Clinical Development
|
February 10, 2016
Targeted approaches to induce immune tolerance for Pompe disease therapy
Phillip A Doerfler, Sushrusha Nayak, Manuela Corti, et al.
Plant Biotechnology Journal
|
June 9, 2015
Oral delivery of Acid Alpha Glucosidase epitopes expressed in plant chloroplasts suppresses antibody formation in treatment of Pompe mice
Jin Su, Alexandra Sherman, Phillip A Doerfler, et al.
The Journal of Clinical Investigation
|
April 15, 2021
Genetic therapies for the first molecular disease
Phillip A Doerfler, Akshay Sharma, Jerlym S Porter, et al.
Journal of Sickle Cell Disease
|
April 30, 2025
Hydroxyurea reduces the levels of the fetal <i>globin</i> gene repressors ZBTB7A/LRF and BCL11A in erythroid cells <i>in vitro</i>
Gabriella E Martyn, Phillip A Doerfler, Yu Yao, et al.
Human Gene Therapy
|
November 26, 2015
Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease
Phillip A Doerfler, Adrian G Todd, Nathalie Clément, et al.
Plos One
|
June 5, 2014
Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent
Sushrusha Nayak, Phillip A Doerfler, Stacy L Porvasnik, et al.
Nature Genetics
|
April 13, 2021
Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing
Mitchell L Leibowitz, Stamatis Papathanasiou, Phillip A Doerfler, et al.
Disease Models & Mechanisms
|
July 6, 2022
Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations
Kaitly J Woodard, Phillip A Doerfler, Kalin D Mayberry, et al.
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of 2
Search research articles
Search
Showing results (1-10 of 18) with videos related to
Sort By:
Page
of 2
Human Gene Therapy Methods
|
August 22, 2014
Copackaging of multiple adeno-associated viral vectors in a single production step
Phillip A Doerfler, Barry J Byrne, Nathalie Clément
Clinical Immunology (Orlando, Fla.)
|
April 6, 2015
BAFF blockade prevents anti-drug antibody formation in a mouse model of Pompe disease
Phillip A Doerfler, Sushrusha Nayak, Roland W Herzog, et al.
Molecular Therapy. Methods & Clinical Development
|
February 10, 2016
Targeted approaches to induce immune tolerance for Pompe disease therapy
Phillip A Doerfler, Sushrusha Nayak, Manuela Corti, et al.
Plant Biotechnology Journal
|
June 9, 2015
Oral delivery of Acid Alpha Glucosidase epitopes expressed in plant chloroplasts suppresses antibody formation in treatment of Pompe mice
Jin Su, Alexandra Sherman, Phillip A Doerfler, et al.
The Journal of Clinical Investigation
|
April 15, 2021
Genetic therapies for the first molecular disease
Phillip A Doerfler, Akshay Sharma, Jerlym S Porter, et al.
Journal of Sickle Cell Disease
|
April 30, 2025
Hydroxyurea reduces the levels of the fetal <i>globin</i> gene repressors ZBTB7A/LRF and BCL11A in erythroid cells <i>in vitro</i>
Gabriella E Martyn, Phillip A Doerfler, Yu Yao, et al.
Human Gene Therapy
|
November 26, 2015
Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease
Phillip A Doerfler, Adrian G Todd, Nathalie Clément, et al.
Plos One
|
June 5, 2014
Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent
Sushrusha Nayak, Phillip A Doerfler, Stacy L Porvasnik, et al.
Nature Genetics
|
April 13, 2021
Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing
Mitchell L Leibowitz, Stamatis Papathanasiou, Phillip A Doerfler, et al.
Disease Models & Mechanisms
|
July 6, 2022
Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations
Kaitly J Woodard, Phillip A Doerfler, Kalin D Mayberry, et al.
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of 2