Adeno-associated virus (AAV) capsid engineering in liver-directed gene therapy

High-Throughput In Vitro, Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction

(2020) Adrian Westhaus et al.   HUMAN GENE THERAPY

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

(2020) Helena Costa Verdera et al.   MOLECULAR THERAPY

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors

(2020) Dan Wang et al.   CELL

Adeno-Associated Virus Genome Interactions Important for Vector Production and Transduction

(2020) Anna C. Maurer et al.   HUMAN GENE THERAPY

COVID-19: Gene Transfer to the Rescue?

(2020) Luk H. Vandenberghe   HUMAN GENE THERAPY

Multiplexed Cre-dependent selection yields systemic AAVs for targeting distinct brain cell types

(2020) Sripriya Ravindra Kumar et al.   NATURE METHODS

Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy

(2020) James M. Wilson et al.   HUMAN GENE THERAPY

AAV Vectors: Are They Safe?

(2020) Arun Srivastava   HUMAN GENE THERAPY

Site-Directed Mutagenesis Improves the Transduction Efficiency of Capsid Library-Derived Recombinant AAV Vectors

(2020) Gai Ran et al.   Molecular Therapy-Methods & Clinical Development

Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2

(2020) Marti Cabanes-Creus et al.   Molecular Therapy-Methods & Clinical Development

In vivo–directed evolution of adeno-associated virus in the primate retina

(2020) Leah C. Byrne et al.   JCI Insight

Development of AAV Variants with Human Hepatocyte Tropism and Neutralizing Antibody Escape Capacity

(2020) Xiaolei Pei et al.   Molecular Therapy-Methods & Clinical Development

Occurrence of the potent mutagens 2- nitrobenzanthrone and 3-nitrobenzanthrone in fine airborne particles

(2019) Aldenor G. Santos et al.   Scientific Reports

Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors

(2019) Hildegard Büning 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

Next Generation of Adeno-Associated Virus Vectors for Gene Therapy for Human Liver Diseases

(2019) Kenneth I. Berns et al.   GASTROENTEROLOGY CLINICS OF NORTH AMERICA

Clinical advances in gene therapy updates on clinical trials of gene therapy in haemophilia

(2019) Flora Peyvandi et al.   HAEMOPHILIA

Genetic based approaches to inherited metabolic liver diseases

(2019) Nerea Zabaleta et al.   HUMAN GENE THERAPY

Advances and challenges for hemophilia gene therapy

(2019) Paul Batty et al.   HUMAN MOLECULAR GENETICS

Gene Therapy

(2019) Katherine A. High et al.   NEW ENGLAND JOURNAL OF MEDICINE

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

(2019) Allison M. Keeler et al.   Annual Review of Virology

Post‐translational modifications in capsid proteins of recombinant adeno‐associated virus ( AAV ) 1‐rh10 serotypes

(2019) Bertin Mary et al.   FEBS Journal

Gene therapy for hemophilia: Progress to date and challenges moving forward

(2019) Kandace L. Gollomp et al.   TRANSFUSION AND APHERESIS SCIENCE

Clinical Considerations for Capsid Choice in the Development of Liver-Targeted AAV-Based Gene Transfer

(2019) Steven Pipe et al.   Molecular Therapy-Methods & Clinical Development

Co-Prevalence of Pre-Existing Immunity to Different Serotypes of Adeno-Associated Virus (AAV) in Adults with Hemophilia

(2019) Kavitha Rajavel et al.   BLOOD

GPR108 Is a Highly Conserved AAV Entry Factor

(2019) Amanda M. Dudek et al.   MOLECULAR THERAPY

Comprehensive AAV capsid fitness landscape reveals a viral gene and enables machine-guided design

(2019) Pierce J. Ogden et al.   SCIENCE

A User's Guide to the Inverted Terminal Repeats of Adeno-Associated Virus

(2019) Patrick Wilmott et al.   Human Gene Therapy Methods

An engineered serum albumin-binding AAV9 capsid achieves improved liver transduction after intravenous delivery in mice

(2019) Quan Jin et al.   GENE THERAPY

Selection of an Efficient AAV Vector for Robust CNS Transgene Expression

(2019) Killian S. Hanlon et al.   Molecular Therapy-Methods & Clinical Development

Past, present and future of haemophilia gene therapy: From vectors and transgenes to known and unknown outcomes

(2018) G. F. Pierce et al.   HAEMOPHILIA

Characterization of AAV vector particle stability at the single-capsid level

(2018) Julien Bernaud et al.   JOURNAL OF BIOLOGICAL PHYSICS

High-Throughput Dissection of AAV–Host Interactions: The Fast and the Curious

(2018) Anne-Kathrin Herrmann et al.   JOURNAL OF MOLECULAR BIOLOGY

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

(2018) Nicole K. Paulk et al.   MOLECULAR THERAPY

Pharmacology of Recombinant Adeno-associated Virus Production

(2018) Magalie Penaud-Budloo et al.   Molecular Therapy-Methods & Clinical Development

Update on clinical gene therapy for hemophilia

(2018) George Q. Perrin et al.   BLOOD

Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye

(2018) Gerard A. Rodrigues et al.   PHARMACEUTICAL RESEARCH

Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors

(2017) Marianna Hösel et al.   HEPATOLOGY

Hemophilia Gene Therapy: Ready for Prime Time?

(2017) Thierry VandenDriessche et al.   HUMAN GENE THERAPY

Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial

(2017) Céline Vandamme et al.   HUMAN GENE THERAPY

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

(2017) Julien Baruteau et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Identification of liver-specific enhancer–promoter activity in the 3′ untranslated region of the wild-type AAV2 genome

(2017) Grant J Logan et al.   NATURE GENETICS

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

(2017) Lindsey A. George et al.   NEW ENGLAND JOURNAL OF MEDICINE

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Liver induced transgene tolerance with AAV vectors

(2017) Geoffrey D. Keeler et al.   CELLULAR IMMUNOLOGY

Complexity of immune responses to AAV transgene products – Example of factor IX

(2017) Roland W. Herzog   CELLULAR IMMUNOLOGY

A Precise Chemical Strategy To Alter the Receptor Specificity of the Adeno-Associated Virus

(2016) Rachel E. Kelemen et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Adeno-Associated Virus Gene Therapy for Liver Disease

(2016) Lisa M. Kattenhorn et al.   HUMAN GENE THERAPY

Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid

(2016) Koen Vercauteren et al.   MOLECULAR THERAPY

An essential receptor for adeno-associated virus infection

(2016) S. Pillay et al.   NATURE

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

Gene therapy for hemophilia: past, present and future

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

Engineering the AAV capsid to optimize vector–host-interactions

(2015) Hildegard Büning et al.   CURRENT OPINION IN PHARMACOLOGY

Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids

(2015) Lili Wang 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

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

Recombinant adeno-associated virus vectors in the treatment of rare diseases

(2015) Eric Hastie et al.   Expert Opinion on Orphan Drugs

Super-resolution imaging of nuclear import of adeno-associated virus in live cells

(2015) Joseph M Kelich et al.   Molecular Therapy-Methods & Clinical Development

Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter

(2014) T. Cronin et al.   EMBO Molecular Medicine

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

The X Gene of Adeno-Associated Virus 2 (AAV2) Is Involved in Viral DNA Replication

(2014) Maohua Cao et al.   PLoS One

Intramuscular Injection of AAV8 in Mice and Macaques Is Associated with Substantial Hepatic Targeting and Transgene Expression

(2014) Jenny A. Greig et al.   PLoS One

Parvovirus glycan interactions

(2014) Lin-Ya Huang et al.   Current Opinion in Virology

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

(2013) A. T. Martino et al.   BLOOD

Gene therapy on the move

(2013) Kerstin B. Kaufmann et al.   EMBO Molecular Medicine

Electron microscopy analysis of a disaccharide analog complex reveals receptor interactions of adeno-associated virus

(2013) Qing Xie et al.   JOURNAL OF STRUCTURAL BIOLOGY

Structure and Dynamics of Adeno-Associated Virus Serotype 1 VP1-Unique N-Terminal Domain and Its Role in Capsid Trafficking

(2013) B. Venkatakrishnan et al.   JOURNAL OF VIROLOGY

Differential Adeno-Associated Virus Serotype-Specific Interaction Patterns with Synthetic Heparins and Other Glycans

(2013) M. Mietzsch et al.   JOURNAL OF VIROLOGY

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

(2013) Leszek Lisowski et al.   NATURE

Comparison of gene transfer to the murine liver following intraperitoneal and intraportal delivery of hepatotropic AAV pseudo-serotypes

(2012) A P Dane et al.   GENE THERAPY

The Threefold Protrusions of Adeno-Associated Virus Type 8 Are Involved in Cell Surface Targeting as Well as Postattachment Processing

(2012) C. Raupp et al.   JOURNAL OF VIROLOGY

Displaying High-affinity Ligands on Adeno-associated Viral Vectors Enables Tumor Cell-specific and Safe Gene Transfer

(2012) Robert C Münch et al.   MOLECULAR THERAPY

Site-Specific Modification of Adeno-Associated Viruses via a Genetically Engineered Aldehyde Tag

(2012) Yarong Liu et al.   Small

The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver

(2011) A. T. Martino et al.   BLOOD

Successful target cell transduction of capsid-engineered rAAV vectors requires clathrin-dependent endocytosis

(2011) S Uhrig et al.   GENE THERAPY

Development of optimized AAV3 serotype vectors: mechanism of high-efficiency transduction of human liver cancer cells

(2011) B Cheng et al.   GENE THERAPY

Toll-like receptor 2-mediated innate immune response in human nonparenchymal liver cells toward adeno-associated viral vectors

(2011) Marianna Hösel et al.   HEPATOLOGY

Stable Human FIX Expression After 0.9G Intrauterine Gene Transfer of Self-complementary Adeno-associated Viral Vector 5 and 8 in Macaques

(2011) Citra NZ Mattar et al.   MOLECULAR THERAPY

Directed Evolution of Adeno-associated Virus for Enhanced Gene Delivery and Gene Targeting in Human Pluripotent Stem Cells

(2011) Prashanth Asuri et al.   MOLECULAR THERAPY

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

Epidermal growth factor receptor is a co-receptor for adeno-associated virus serotype 6

(2010) Melodie L Weller et al.   NATURE MEDICINE

A viral assembly factor promotes AAV2 capsid formation in the nucleolus

(2010) F. Sonntag et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Intrinsic phospholipase A2 activity of adeno-associated virus is involved in endosomal escape of incoming particles

(2010) Stefanie Stahnke et al.   VIROLOGY

Engineering adeno-associated virus serotype 2-based targeting vectors using a new insertion site-position 453-and single point mutations

(2009) Jorge Boucas et al.   JOURNAL OF GENE MEDICINE

Molecular Evolution of Adeno-associated Virus for Enhanced Glial Gene Delivery

(2009) James T Koerber et al.   MOLECULAR THERAPY

Immunomodulatory Gene Therapy Prevents Antibody Formation and Lethal Hypersensitivity Reactions in Murine Pompe Disease

(2009) Baodong Sun et al.   MOLECULAR THERAPY

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

(2008) William W. Hauswirth et al.   HUMAN GENE THERAPY

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

(2008) D. Grimm et al.   JOURNAL OF VIROLOGY

DNA Shuffling of Adeno-associated Virus Yields Functionally Diverse Viral Progeny

(2008) James T Koerber et al.   MOLECULAR THERAPY

Self-complementary AAV Vectors; Advances and Applications

(2008) Douglas M McCarty   MOLECULAR THERAPY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

(2008) A. V. Cideciyan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Optimization of Self-complementary AAV Vectors for Liver-directed Expression Results in Sustained Correction of Hemophilia B at Low Vector Dose

(2007) Zhijian Wu et al.   MOLECULAR THERAPY