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Gene Therapy
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July 1, 2022
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials
Liching Cao, Annemarie Ledeboer, Yonghua Pan, et al.
The Journal of Pharmacology and Experimental Therapeutics
|
November 19, 2015
A Novel Allosteric Insulin Receptor-Activating Antibody Reduces Hyperglycemia without Hypoglycemia in Diabetic Cynomolgus Monkeys
Padma Bezwada, Jingsong Zhao, Ken Der, et al.
Molecular Therapy. Methods & Clinical Development
|
November 3, 2023
A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies
Yonghua Pan, Michelle Rohde, Jennifer Zeitler, et al.
Nucleic Acids Research
|
September 10, 2004
Multiplex mRNA assay using electrophoretic tags for high-throughput gene expression analysis
Huan Tian, Liching Cao, Yuping Tan, et al.
Molecular Therapy. Methods & Clinical Development
|
August 11, 2020
AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction
Makiko Yasuda, Marshall W Huston, Silvere Pagant, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 27, 2022
First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B
Paul Harmatz, Carlos E Prada, Barbara K Burton, et al.
Blood
|
October 23, 2023
Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study
Andrew D Leavitt, Barbara A Konkle, Kimo C Stine, et al.
The AAPS Journal
|
September 4, 2024
Correction to: Neutralizing Antibody Sample Testing and Report Harmonization
Darshana Jani, Michele Gunsior, Robin Marsden, et al.
The AAPS Journal
|
July 11, 2024
Neutralizing Antibody Sample Testing and Report Harmonization
Darshana Jani, Michele Gunsior, Robin Marsden, et al.
The AAPS Journal
|
October 28, 2022
Anti-drug Antibody Sample Testing and Reporting Harmonization
Darshana Jani, Robin Marsden, Michele Gunsior, et al.
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of 2
Search research articles
Search
Showing results (1-10 of 13) with videos related to
Sort By:
Page
of 2
Gene Therapy
|
July 1, 2022
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials
Liching Cao, Annemarie Ledeboer, Yonghua Pan, et al.
The Journal of Pharmacology and Experimental Therapeutics
|
November 19, 2015
A Novel Allosteric Insulin Receptor-Activating Antibody Reduces Hyperglycemia without Hypoglycemia in Diabetic Cynomolgus Monkeys
Padma Bezwada, Jingsong Zhao, Ken Der, et al.
Molecular Therapy. Methods & Clinical Development
|
November 3, 2023
A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies
Yonghua Pan, Michelle Rohde, Jennifer Zeitler, et al.
Nucleic Acids Research
|
September 10, 2004
Multiplex mRNA assay using electrophoretic tags for high-throughput gene expression analysis
Huan Tian, Liching Cao, Yuping Tan, et al.
Molecular Therapy. Methods & Clinical Development
|
August 11, 2020
AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction
Makiko Yasuda, Marshall W Huston, Silvere Pagant, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 27, 2022
First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B
Paul Harmatz, Carlos E Prada, Barbara K Burton, et al.
Blood
|
October 23, 2023
Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study
Andrew D Leavitt, Barbara A Konkle, Kimo C Stine, et al.
The AAPS Journal
|
September 4, 2024
Correction to: Neutralizing Antibody Sample Testing and Report Harmonization
Darshana Jani, Michele Gunsior, Robin Marsden, et al.
The AAPS Journal
|
July 11, 2024
Neutralizing Antibody Sample Testing and Report Harmonization
Darshana Jani, Michele Gunsior, Robin Marsden, et al.
The AAPS Journal
|
October 28, 2022
Anti-drug Antibody Sample Testing and Reporting Harmonization
Darshana Jani, Robin Marsden, Michele Gunsior, et al.
Page
of 2