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Human Gene Therapy|June 25, 2020
Engineered Capsids for Efficient Gene Delivery to the Retina and CorneaAmy Frederick, Jennifer Sullivan, Lin Liu, et al.
Human Gene Therapy|September 6, 2021
Improved Potency and Safety of DNA-Encoded Antibody Therapeutics Through Plasmid Backbone and Expression Cassette EngineeringGiles Vermeire, Elien De Smidt, Nick Geukens, et al.
Human Gene Therapy|August 11, 2021
Development and Application of a Liquid Chromatography-Mass Spectrometry Method for Residual Iodixanol Quantification in AAV-Based Gene Therapy Product DevelopmentYi Pu, Russell Katz, Yunqiu Chen, et al.
Human Gene Therapy|September 9, 2021
Preclinical Assessment of a Gene-Editing Approach in a Mouse Model of Mitochondrial Neurogastrointestinal EncephalomyopathyMarta Parés, Cristina Fornaguera, Ferran Vila-Julià, et al.
Human Gene Therapy|September 8, 2021
Recent Progress in Genome Editing for Gene Therapy Applications: The French PerspectiveMario Amendola, Aurélie Bedel, Ana Buj-Bello, et al.
Human Gene Therapy|August 18, 2021
Induction of Robust Type I Interferon Levels by Oncolytic Reovirus Requires Both Viral Replication and Interferon-α/β Receptor SignalingTimo Oosenbrug, Diana J M van den Wollenberg, Eline W Duits, et al.
Human Gene Therapy|February 10, 2023
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular AtrophyYueyi Li, Hongyu Zeng, Yuhao Wei, et al.
Human Gene Therapy|March 23, 2023
Characterization of a Bioengineered AAV3B Capsid Variant with Enhanced Hepatocyte Tropism and Immune EvasionJyoti Rana, Damien Marsic, Chenhui Zou, et al.
Human Gene Therapy|November 14, 2022
Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of MeganucleasesJenny A Greig, Camilo Breton, Scott N Ashley, et al.
Human Gene Therapy|November 19, 2022
Process Development of Recombinant Adeno-Associated Virus Production Platform Results in High Production Yield and PurityHuiren Zhao, W Hans Meisen, Songli Wang, et al.
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