Improved adeno-associated virus (AAV) serotype 1 and 5 vectors for gene therapy

Targeted Modifications in Adeno-Associated Virus Serotype 8 Capsid Improves Its Hepatic Gene Transfer Efficiency In Vivo

(2013) Dwaipayan Sen et al.   Human Gene Therapy Methods

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

The gene therapy journey for hemophilia: are we there yet?

(2012) K. A. High   BLOOD

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

(2012) G. Buchlis et al.   BLOOD

AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice

(2012) C Hu et al.   GENE THERAPY

Directed evolution of novel adeno-associated viruses for therapeutic gene delivery

(2012) M A Bartel et al.   GENE THERAPY

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

(2012) J. Bennett et al.   Science Translational Medicine

Universal Real-Time PCR for the Detection and Quantification of Adeno-Associated Virus Serotype 2-Derived Inverted Terminal Repeat Sequences

(2012) Christine Aurnhammer et al.   Human Gene Therapy Methods

Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia

(2011) C Li et al.   GENE THERAPY

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

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

Sites in the AAV5 capsid tolerant to deletions and tandem duplications

(2010) Kaoru Hida et al.   ARCHIVES OF BIOCHEMISTRY AND BIOPHYSICS

Comparative Cardiac Gene Delivery of Adeno-Associated Virus Serotypes 1-9 reveals that AAV6 Mediates the Most Efficient Transduction in Mouse Heart

(2010) Carmela Zincarelli et al.   CTS-Clinical and Translational Science

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

Effect of Adeno-Associated Virus Serotype and Genomic Structure on Liver Transduction and Biodistribution in Mice of Both Genders

(2009) Astrid Pañeda et al.   HUMAN GENE THERAPY

Comparison of Adeno-Associated Virus Pseudotype 1, 2, and 8 Vectors Administered by Intramuscular Injection in the Treatment of Murine Phenylketonuria

(2009) Alexandre Rebuffat et al.   HUMAN GENE THERAPY

Adeno-associated virus serotype 2 induces cell-mediated immune responses directed against multiple epitopes of the capsid protein VP1

(2009) D. Madsen et al.   JOURNAL OF GENERAL VIROLOGY

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Comparative Transduction Efficiency of AAV Vector Serotypes 1–6 in the Substantia Nigra and Striatum of the Primate Brain

(2009) Eleni A Markakis et al.   MOLECULAR THERAPY

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

(2009) Jonathan D Finn et al.   MOLECULAR THERAPY

Gene Therapy Using Adeno-Associated Virus Vectors

(2008) S. Daya et al.   CLINICAL MICROBIOLOGY REVIEWS

Transient Immunomodulation Allows Repeated Injections of AAV1 and Correction of Muscular Dystrophy in Multiple Muscles

(2008) Stéphanie Lorain et al.   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

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

Prolonged Susceptibility to Antibody-mediated Neutralization for Adeno-associated Vectors Targeted to the Liver

(2007) Samuel L Murphy et al.   MOLECULAR THERAPY