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Phillip A Doerfler

Showing results (1-10 of 18) with videos related to

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Human Gene Therapy Methods|August 22, 2014
Copackaging of multiple adeno-associated viral vectors in a single production stepPhillip 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 diseasePhillip 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 therapyPhillip 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 miceJin Su, Alexandra Sherman, Phillip A Doerfler, et al.
The Journal of Clinical Investigation|April 15, 2021
Genetic therapies for the first molecular diseasePhillip 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 DiseasePhillip 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 dependentSushrusha Nayak, Phillip A Doerfler, Stacy L Porvasnik, et al.
Nature Genetics|April 13, 2021
Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editingMitchell 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 configurationsKaitly J Woodard, Phillip A Doerfler, Kalin D Mayberry, et al.
Pageof 2

Showing results (1-10 of 18) with videos related to

Sort By:
Pageof 2
Human Gene Therapy Methods|August 22, 2014
Copackaging of multiple adeno-associated viral vectors in a single production stepPhillip 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 diseasePhillip 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 therapyPhillip 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 miceJin Su, Alexandra Sherman, Phillip A Doerfler, et al.
The Journal of Clinical Investigation|April 15, 2021
Genetic therapies for the first molecular diseasePhillip 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 DiseasePhillip 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 dependentSushrusha Nayak, Phillip A Doerfler, Stacy L Porvasnik, et al.
Nature Genetics|April 13, 2021
Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editingMitchell 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 configurationsKaitly J Woodard, Phillip A Doerfler, Kalin D Mayberry, et al.
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