AAV integration in human hepatocytes

Hepatitis B virus integrations promote local and distant oncogenic driver alterations in hepatocellular carcinoma

(2021) Camille Péneau et al.   GUT

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

(2020) Helena Costa Verdera et al.   MOLECULAR THERAPY

Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking

(2020) Samuele Ferrari et al.   NATURE BIOTECHNOLOGY

Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing

(2020) Samantha G. Scharenberg et al.   Nature Communications

Liver injury increases incidence of HCC following AAV gene therapy in mice

(2020) Dhwanil A. Dalwadi et al.   MOLECULAR THERAPY

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

Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors

(2020) Rita Ferla et al.   Molecular Therapy-Methods & Clinical Development

Adeno-associated Virus (AAV) versus Immune Response

(2019) Joseph Rabinowitz et al.   Viruses-Basel

Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response

(2019) Giulia Schiroli et al.   Cell Stem Cell

Adeno-associated virus in the liver: natural history and consequences in tumour development

(2019) Tiziana La Bella et al.   GUT

Intrathymic adeno-associated virus gene transfer rapidly restores thymic function and long-term persistence of gene-corrected T cells

(2019) Marie Pouzolles et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders

(2019) Alireza Shahryari et al.   Frontiers in Genetics

Long-Term Correction of Diabetes in Mice by In Vivo Reprogramming of Pancreatic Ducts

(2018) Yuhan Wang et al.   MOLECULAR THERAPY

Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras

(2018) Phillip W.L. Tai et al.   Molecular Therapy-Methods & Clinical Development

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

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

Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector–mediated intrauterine gene transfer in early-gestation fetal macaques

(2018) Jerry K. Y. Chan et al.   FASEB JOURNAL

Entering the Modern Era of Gene Therapy

(2018) Xavier M. Anguela et al.   Annual Review of Medicine

Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models

(2017) Randy J. Chandler et al.   HUMAN GENE THERAPY

Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity

(2017) Jun Xie et al.   MOLECULAR THERAPY

In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model

(2017) Citra N.Z. Mattar et al.   MOLECULAR THERAPY

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

(2017) Giulia Schiroli et al.   Science Translational Medicine

Persistent Expression of Dopamine-Synthesizing Enzymes 15 Years After Gene Transfer in a Primate Model of Parkinson's Disease

(2017) Yoshihide Sehara et al.   Human Gene Therapy Clinical Development

MultiQC: summarize analysis results for multiple tools and samples in a single report

(2016) Philip Ewels et al.   BIOINFORMATICS

Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria

(2016) Delia D’Avola et al.   JOURNAL OF HEPATOLOGY

Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?

(2016) Randy J Chandler et al.   MOLECULAR THERAPY

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

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

Interaction of vectors and parental viruses with the host genome

(2016) Irene Gil-Farina et al.   Current Opinion in Virology

In vivo tissue-tropism of adeno-associated viral vectors

(2016) Arun Srivastava   Current Opinion in Virology

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

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

Long-Term Correction of Sandhoff Disease Following Intravenous Delivery of rAAV9 to Mouse Neonates

(2015) Jagdeep S Walia et al.   MOLECULAR THERAPY

Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors

(2015) Jianbin Wang et al.   NATURE BIOTECHNOLOGY

Adeno-associated virus finds its disease

(2015) David W Russell et al.   NATURE GENETICS

Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas

(2015) Jean-Charles Nault et al.   NATURE GENETICS

Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

(2015) Blythe D. Sather et al.   Science Translational Medicine

Extensive double humanization of both liver and hematopoiesis in FRGN mice

(2014) Elizabeth M. Wilson et al.   Stem Cell Research

VISPA: a computational pipeline for the identification and analysis of genomic vector integration sites

(2014) Andrea Calabria et al.   Genome Medicine

No tumour-initiating risk associated with scAAV transduction in newborn rat liver

(2013) V Gauttier et al.   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

A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype

(2013) Brian L Ellis et al.   Virology Journal

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

(2012) Lucia E Rosas et al.   MOLECULAR THERAPY

AAV Vectors Containing rDNA Homology Display Increased Chromosomal Integration and Transgene Persistence

(2012) Zhongya Wang et al.   MOLECULAR THERAPY

Induction of hepatocellular carcinoma by in vivo gene targeting

(2012) P.-R. Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

BEDTools: a flexible suite of utilities for comparing genomic features

(2010) Aaron R. Quinlan et al.   BIOINFORMATICS

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

(2010) Nathalia Holt et al.   NATURE BIOTECHNOLOGY

GREAT improves functional interpretation of cis-regulatory regions

(2010) Cory Y McLean et al.   NATURE BIOTECHNOLOGY

Fast and accurate short read alignment with Burrows-Wheeler transform

(2009) H. Li et al.   BIOINFORMATICS

MEME SUITE: tools for motif discovery and searching

(2009) T. L. Bailey et al.   NUCLEIC ACIDS RESEARCH

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

Gene Therapy Using Adeno-Associated Virus Vectors

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

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

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

Recombinant Adeno-associated Virus Transduction and Integration

(2008) Brian R Schultz et al.   MOLECULAR THERAPY

Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: Effects of serotype and site of administration

(2007) Ji-jing Pang et al.   VISION RESEARCH