Integrated vector genomes may contribute to long-term expression in primate liver after AAV administration

Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A

(2022) Sylvia Fong et al.   NATURE MEDICINE

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

(2022) Margareth C. Ozelo et al.   NEW ENGLAND JOURNAL OF MEDICINE

Integrative proteogenomic characterization of hepatocellular carcinoma across etiologies and stages

(2022) Charlotte K. Y. Ng et al.   Nature Communications

Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression

(2022) Lili Wang et al.   Molecular Therapy-Methods & Clinical Development

The gut virome: A new microbiome component in health and disease

(2022) Zhirui Cao et al.   EBioMedicine

HDAC inhibition prevents transgene expression downregulation and loss of function in T cell receptor-transduced T cells

(2021) Tamson V. Moore et al.   Molecular Therapy-Oncolytics

Integrated analysis of multimodal single-cell data

(2021) Yuhan Hao et al.   CELL

AAV integration in human hepatocytes

(2021) Dhwanil A. Dalwadi et al.   MOLECULAR THERAPY

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A

(2021) Lindsey A. George et al.   NEW ENGLAND JOURNAL OF MEDICINE

ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing

(2020) Camilo Breton et al.   BMC GENOMICS

BAX 335 hemophilia B gene therapy clinical trial results - potential impact of CpG sequences on gene expression

(2020) Barbara A Konkle et al.   BLOOD

A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells

(2020) Giang N. Nguyen et al.   NATURE BIOTECHNOLOGY

Clinical impact of genomic diversity from early to advanced hepatocellular carcinoma

(2019) Jean-Charles Nault et al.   HEPATOLOGY

Optimized Adeno-Associated Virus-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques

(2018) Jenny Greig et al.   HUMAN GENE THERAPY

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

(2018) Pasqualina Colella et al.   Molecular Therapy-Methods & Clinical Development

Spatiotemporal transcriptomic divergence across human and macaque brain development

(2018) Ying Zhu et al.   SCIENCE

AAV8 Gene Therapy for Crigler-Najjar Syndrome in Macaques Elicited Transgene T Cell Responses That Are Resident to the Liver

(2018) Jenny A. Greig et al.   Molecular Therapy-Methods & Clinical Development

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

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

Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR+/− Rhesus Macaques

(2017) Jenny A. Greig et al.   Human Gene Therapy Clinical Development

Recombinant AAV Integration Is Not Associated With Hepatic Genotoxicity in Nonhuman Primates and Patients

(2016) Irene Gil-Farina et al.   MOLECULAR THERAPY

Viral evasion of intracellular DNA and RNA sensing

(2016) Ying Kai Chan et al.   NATURE REVIEWS MICROBIOLOGY

Impact of intravenous infusion time on AAV8 vector pharmacokinetics, safety, and liver transduction in cynomolgus macaques

(2016) Jenny A Greig et al.   Molecular Therapy-Methods & Clinical Development

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy

(2015) Randy J. Chandler et al.   JOURNAL OF CLINICAL INVESTIGATION

Adeno-Associated Virus Type 2 Wild-Type and Vector-Mediated Genomic Integration Profiles of Human Diploid Fibroblasts Analyzed by Third-Generation PacBio DNA Sequencing

(2014) D. Huser et al.   JOURNAL OF VIROLOGY

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

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

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

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

(2013) J. McIntosh et al.   BLOOD

Recombinant Adeno-Associated Virus Integration Sites in Murine Liver After Ornithine Transcarbamylase Gene Correction

(2013) Li Zhong et al.   HUMAN GENE THERAPY

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

(2013) Astrid Pañeda et al.   HUMAN GENE THERAPY

A largely random AAV integration profile after LPLD gene therapy

(2013) Christine Kaeppel et al.   NATURE MEDICINE

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

Patterns of scAAV Vector Insertion Associated With Oncogenic Events in a Mouse Model for Genotoxicity

(2012) Lucia E Rosas 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

Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy

(2010) Gregory D Hurlbut et al.   MOLECULAR THERAPY

Pathogenesis of hepatitis B virus infection

(2010) F.V. Chisari et al.   PATHOLOGIE BIOLOGIE

Stem Cells and Liver Regeneration

(2009) Andrew W. Duncan et al.   GASTROENTEROLOGY

Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo

(2009) Nicole K. Paulk et al.   HEPATOLOGY

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

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

Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

(2008) Sharon C Cunningham et al.   MOLECULAR THERAPY