Safety and Liver Transduction Efficacy of rAAV5-cohPBGD in Nonhuman Primates: A Potential Therapy for Acute Intermittent Porphyria

A largely random AAV integration profile after LPLD gene therapy

(2013) Christine Kaeppel et al.   NATURE MEDICINE

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

(2013) Dwaipayan Sen et al.   Scientific Reports

Integration Frequency and Intermolecular Recombination of rAAV Vectors in Non-human Primate Skeletal Muscle and Liver

(2012) Ali Nowrouzi et al.   MOLECULAR THERAPY

Mutation in the platelet-derived growth factor receptor alpha inhibits adeno-associated virus type 5 transduction

(2012) Ingo H. Pilz et al.   VIROLOGY

Systemic Errors in Quantitative Polymerase Chain Reaction Titration of Self-Complementary Adeno-Associated Viral Vectors and Improved Alternative Methods

(2012) Paolo Fagone et al.   Human Gene Therapy Methods

Immune Responses to AAV in Clinical Trials

(2011) Federico Mingozzi et al.   CURRENT GENE THERAPY

Adeno-Associated Virus Liver Transduction Efficiency Measured by in Vivo [18F]FHBG Positron Emission Tomography Imaging in Rodents and Nonhuman Primates

(2011) Astrid Pañeda et al.   HUMAN GENE THERAPY

Liver transplantation for acute intermittent porphyria is complicated by a high rate of hepatic artery thrombosis

(2011) Joanna K. Dowman et al.   LIVER TRANSPLANTATION

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

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

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

Analyzing the Number of Common Integration Sites of Viral Vectors – New Methods and Computer Programs

(2011) Ulrich Abel et al.   PLoS One

Assessing the potential for AAV vector genotoxicity in a murine model

(2010) H. Li et al.   BLOOD

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

Sustained Enzymatic Correction by rAAV-Mediated Liver Gene Therapy Protects Against Induced Motor Neuropathy in Acute Porphyria Mice

(2010) Carmen Unzu et al.   MOLECULAR THERAPY

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

(2010) Anna Paruzynski et al.   Nature Protocols

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

Porphobilinogen deaminase over-expression in hepatocytes, but not in erythrocytes, prevents accumulation of toxic porphyrin precursors in a mouse model of acute intermittent porphyria

(2009) Carmen Unzu et al.   JOURNAL OF HEPATOLOGY

Host and Vector-dependent Effects on the Risk of Germline Transmission of AAV Vectors

(2009) Patricia Favaro et al.   MOLECULAR THERAPY

AAV8-mediated Gene Therapy Prevents Induced Biochemical Attacks of Acute Intermittent Porphyria and Improves Neuromotor Function

(2009) Makiko Yasuda et al.   MOLECULAR THERAPY

Comprehensive genomic access to vector integration in clinical gene therapy

(2009) Richard Gabriel et al.   NATURE MEDICINE