Assessment of the percentage of full recombinant adeno-associated virus particles in a gene therapy drug using CryoTEM

Comprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering

(2021) Nicole L. McIntosh et al.   Scientific Reports

Quantitative Analysis of Genome Packaging in Recombinant AAV Vectors by Charge Detection Mass Spectrometry

(2021) Lauren F. Barnes et al.   Molecular Therapy-Methods & Clinical Development

Comparison of Analytical Techniques to Quantitate the Capsid Content of Adeno-Associated Viral Vectors

(2021) Amanda K. Werle et al.   Molecular Therapy-Methods & Clinical Development

Assessment of genome packaging in AAVs using Orbitrap-based charge-detection mass spectrometry

(2021) Tobias P. Wörner et al.   Molecular Therapy-Methods & Clinical Development

Development of an In Vitro Biopotency Assay for an AAV8 Hemophilia B Gene Therapy Vector Suitable for Clinical Product Release

(2020) Johannes Lengler et al.   Molecular Therapy-Methods & Clinical Development

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

Gene therapy of hematological disorders: current challenges

(2019) Amr M. Al-Saif   GENE THERAPY

Filling AAV capsids: estimating success by cryo-EM

(2019) Suriyasri Subramanian et al.   HUMAN GENE THERAPY

Highly Efficient Ultracentrifugation-free Chromatographic Purification of Recombinant AAV Serotype 9

(2018) Taro Tomono et al.   Molecular Therapy-Methods & Clinical Development

Entering the Modern Era of Gene Therapy

(2018) Xavier M. Anguela et al.   Annual Review of Medicine

Empty Capsids and Macrophage Inhibition/Depletion Increase rAAV Transgene Expression in Joints of Both Healthy and Arthritic Mice

(2017) Caroline J. Aalbers et al.   HUMAN GENE THERAPY

Empty Adeno-Associated Virus Capsids: Contaminant or Natural Decoy?

(2017) Terence R. Flotte   HUMAN GENE THERAPY

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

(2017) Federico Mingozzi et al.   Annual Review of Virology

Gene therapy for hemophilia: past, present and future

(2016) Lindsey A. George et al.   SEMINARS IN HEMATOLOGY

Practical utilization of recombinant AAV vector reference standards: focus on vector genomes titration by free ITR qPCR

(2016) Susan D'Costa et al.   Molecular Therapy-Methods & Clinical Development

Analytical Ultracentrifugation as an Approach to Characterize Recombinant Adeno-Associated Viral Vectors

(2015) Brenda Burnham et al.   Human Gene Therapy Methods

Definitions in hemophilia: communication from the SSC of the ISTH

(2014) V. S. Blanchette et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

AAV Empty Capsids: For Better or for Worse?

(2014) J Fraser Wright   MOLECULAR THERAPY

Empty virions in AAV8 vector preparations reduce transduction efficiency and may cause total viral particle dose-limiting side effects

(2014) Kai Gao et al.   Molecular Therapy-Methods & Clinical Development

Preclinical safety and efficacy of a new recombinant FIX drug product for treatment of hemophilia B

(2013) Barbara Dietrich et al.   INTERNATIONAL JOURNAL OF HEMATOLOGY

Defining Severity of Hemophilia: More than Factor Levels

(2013) Anna Pavlova et al.   SEMINARS IN THROMBOSIS AND HEMOSTASIS

Virus-mediated gene delivery for human gene therapy

(2012) Mauro Giacca et al.   JOURNAL OF CONTROLLED RELEASE

Analysis of Particle Content of Recombinant Adeno-Associated Virus Serotype 8 Vectors by Ion-Exchange Chromatography

(2012) Martin Lock et al.   Human Gene Therapy Methods

Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial

(2011) James A. Allay et al.   HUMAN GENE THERAPY