Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models

Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy

(2017) Hildegund C.J. Ertl et al.   HUMAN GENE THERAPY

Adeno-Associated Virus: The Naturally Occurring Virus Versus the Recombinant Vector

(2016) Arun Srivastava   HUMAN GENE THERAPY

Canine models of inherited bleeding disorders in the development of coagulation assays, novel protein replacement and gene therapies

(2016) T. C. Nichols et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid

(2016) Koen Vercauteren et al.   MOLECULAR THERAPY

A Cure For Hemophilia: the Promise Becomes a Reality

(2016) Roland W Herzog   MOLECULAR THERAPY

Early clinical data raise the bar for hemophilia gene therapies

(2016) Elie Dolgin   NATURE BIOTECHNOLOGY

Development of Optimized AAV Serotype Vectors for High-Efficiency Transduction at Further Reduced Doses

(2016) Chen Ling et al.   Human Gene Therapy Methods

Clinical development of gene therapy: results and lessons from recent successes

(2016) Sandeep RP Kumar et al.   Molecular Therapy-Methods & Clinical Development

Strategies to generate high-titer, high-potency recombinant AAV3 serotype vectors

(2016) Chen Ling et al.   Molecular Therapy-Methods & Clinical Development

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

(2015) J. M. Crudele et al.   BLOOD

E Pluribus Unum: 50 Years of Research, Millions of Viruses, and One Goal—Tailored Acceleration of AAV Evolution

(2015) Dirk Grimm et al.   MOLECULAR THERAPY

Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors

(2015) Shaoyong Li et al.   MOLECULAR THERAPY

Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model

(2015) Beatrice Bissig-Choisat et al.   Nature Communications

Site-Directed Mutagenesis of Surface-Exposed Lysine Residues Leads to Improved Transduction by AAV2, But Not AAV8, Vectors in Murine HepatocytesIn Vivo

(2015) Baozheng Li et al.   Human Gene Therapy Methods

Translational Data from Adeno-Associated Virus-Mediated Gene Therapy of Hemophilia B in Dogs

(2015) Timothy C. Nichols et al.   Human Gene Therapy Clinical Development

Selective In Vivo Targeting of Human Liver Tumors by Optimized AAV3 Vectors in a Murine Xenograft Model

(2014) Chen Ling et al.   HUMAN GENE THERAPY

Vector Design Tour de Force: Integrating Combinatorial and Rational Approaches to Derive Novel Adeno-associated Virus Variants

(2014) Damien Marsic et al.   MOLECULAR THERAPY

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Ex vivo expanded autologous polyclonal regulatory T cells suppress inhibitor formation in hemophilia

(2014) Debalina Sarkar et al.   Molecular Therapy-Methods & Clinical Development

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells

(2013) A. T. Martino et al.   BLOOD

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

(2013) David M. Markusic et al.   EMBO Molecular Medicine

Adeno-associated virus capsid antigen presentation is dependent on endosomal escape

(2013) Chengwen Li et al.   JOURNAL OF CLINICAL INVESTIGATION

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

(2013) Leszek Lisowski et al.   NATURE

Optimization of the Capsid of Recombinant Adeno-Associated Virus 2 (AAV2) Vectors: The Final Threshold?

(2013) George V. Aslanidi et al.   PLoS One

Bioengineering of AAV2 Capsid at Specific Serine, Threonine, or Lysine Residues Improves Its Transduction Efficiency in Vitro and in Vivo

(2013) Nishanth Gabriel et al.   Human Gene Therapy Methods

Sensitivity of whole blood clotting time and activated partial thromboplastin time for factor IX: relevance to gene therapy and determination of post-transfusion elimination time of canine factor IX in hemophilia B dogs

(2011) T. C. NICHOLS et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

High-efficiency Transduction and Correction of Murine Hemophilia B Using AAV2 Vectors Devoid of Multiple Surface-exposed Tyrosines

(2010) David M Markusic et al.   MOLECULAR THERAPY

Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application

(2010) Paul E Monahan et al.   MOLECULAR THERAPY

Efficacy and Safety of Long-term Prophylaxis in Severe Hemophilia A Dogs Following Liver Gene Therapy Using AAV Vectors

(2010) Denise E Sabatino et al.   MOLECULAR THERAPY

Improved Induction of Immune Tolerance to Factor IX by Hepatic AAV-8 Gene Transfer

(2009) Mario Cooper et al.   HUMAN GENE THERAPY

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

(2009) Gary C. Pien et al.   JOURNAL OF CLINICAL INVESTIGATION

Proteasome Inhibitors Decrease AAV2 Capsid derived Peptide Epitope Presentation on MHC Class I Following Transduction

(2009) Jonathan D Finn et al.   MOLECULAR THERAPY

Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy

(2008) G. P. Niemeyer et al.   BLOOD

Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

(2008) L. Zhong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA