Safety profile of recombinant adeno-associated viral vectors: focus on alipogene tiparvovec (Glybera®)

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

(2013) F. Mingozzi et al.   BLOOD

Gene therapy enters the pharma market: The short story of a long journey

(2013) Hildegard Büning   EMBO Molecular Medicine

A largely random AAV integration profile after LPLD gene therapy

(2013) Christine Kaeppel et al.   NATURE MEDICINE

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

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

Not Reinventing the Wheel: Applying the 3Rs Concepts to Viral Vector Gene Therapy Biodistribution Studies

(2013) Timothy K. MacLachlan et al.   Human Gene Therapy Clinical Development

Lessons Learned from the Clinical Development and Market Authorization of Glybera

(2013) Laura M. Bryant et al.   Human Gene Therapy Clinical Development

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

(2012) D Gaudet et al.   GENE THERAPY

Effect of Alipogene Tiparvovec (AAV1-LPLS447X) on Postprandial Chylomicron Metabolism in Lipoprotein Lipase-Deficient Patients

(2012) André C. Carpentier et al.   JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

The AAV Vector Toolkit: Poised at the Clinical Crossroads

(2012) Aravind Asokan et al.   MOLECULAR THERAPY

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors

(2011) K Varadi et al.   GENE THERAPY

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results

(2011) Terence R. Flotte et al.   HUMAN GENE THERAPY

Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors

(2011) L. WANG et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Hepatocellular carcinoma in a research subject with ornithine transcarbamylase deficiency

(2011) James M. Wilson et al.   MOLECULAR GENETICS AND METABOLISM

Engineering Liver-detargeted AAV9 Vectors for Cardiac and Musculoskeletal Gene Transfer

(2011) Nagesh Pulicherla et al.   MOLECULAR THERAPY

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

(2011) Amit C Nathwani et al.   MOLECULAR THERAPY

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

(2011) Dawn E Bowles 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

Assessing the potential for AAV vector genotoxicity in a murine model

(2010) H. Li et al.   BLOOD

Effect of Administration Route on the Biodistribution and Shedding of Replication-Deficient AAV2: A Qualitative Modelling Approach

(2010) Esther F.A. Brandon et al.   CURRENT GENE THERAPY

Adeno-Associated Virus Serotype 6 Capsid Tyrosine-to-Phenylalanine Mutations Improve Gene Transfer to Skeletal Muscle

(2010) Chunping Qiao et al.   HUMAN GENE THERAPY

Molecular Analysis of Vector Genome Structures After Liver Transduction by Conventional and Self-Complementary Adeno-Associated Viral Serotype Vectors in Murine and Nonhuman Primate Models

(2010) Xun Sun et al.   HUMAN GENE THERAPY

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

The Complex and Evolving Story of T cell Activation to AAV Vector-encoded Transgene Products

(2010) Lauren E Mays et al.   MOLECULAR THERAPY

Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing

(2010) Anna Paruzynski et al.   Nature Protocols

Integration Preferences of Wildtype AAV-2 for Consensus Rep-Binding Sites at Numerous Loci in the Human Genome

(2010) Daniela Hüser et al.   PLoS Pathogens

Limb-girdle muscular dystrophy type 2D gene therapy restores α-sarcoglycan and associated proteins

(2009) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

(2009) F. Mingozzi et al.   BLOOD

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

Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID Trial), a First-in-Human Phase 1/2 Clinical Trial

(2009) Brian E. Jaski et al.   JOURNAL OF CARDIAC FAILURE

Transient expression of genes delivered to newborn rat liver using recombinant adeno-associated virus 2/8 vectors

(2009) Maude Flageul et al.   JOURNAL OF GENE MEDICINE

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

(2009) Albert M Maguire et al.   LANCET

Comprehensive genomic access to vector integration in clinical gene therapy

(2009) Richard Gabriel et al.   NATURE MEDICINE

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

(2009) M. L. Brantly et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Intramuscular Administration of AAV1-Lipoprotein Lipase S447X Lowers Triglycerides in Lipoprotein Lipase–Deficient Patients

(2008) Erik S. Stroes et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

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

Silencing of T lymphocytes by antigen-driven programmed death in recombinant adeno-associated virus vector-mediated gene therapy

(2008) V. M. Velazquez et al.   BLOOD

Stable Integration of Recombinant Adeno-Associated Virus Vector Genomes After Transduction of Murine Hematopoietic Stem Cells

(2008) Zongchao Han et al.   HUMAN GENE THERAPY

Frequency and Spectrum of Genomic Integration of Recombinant Adeno-Associated Virus Serotype 8 Vector in Neonatal Mouse Liver

(2008) K. Inagaki et al.   JOURNAL OF VIROLOGY

Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle

(2008) M. Penaud-Budloo et al.   JOURNAL OF VIROLOGY

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

(2008) Stéphanie Lorain 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