Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease

(2021) Yedda Li et al.   MOLECULAR THERAPY

The clinical landscape for AAV gene therapies

(2021) Dmitry A. Kuzmin et al.   NATURE REVIEWS DRUG DISCOVERY

Hepatocellular carcinoma

(2021) Josep M. Llovet et al.   Nature Reviews Disease Primers

Liver gene therapy and hepatocellular carcinoma: A complex web

(2021) Ype P. de Jong et al.   MOLECULAR THERAPY

AAV integration in human hepatocytes

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

Flies in the ointment: AAV vector preparations and tumor risk

(2021) Junping Zhang et al.   MOLECULAR THERAPY

Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning

(2021) Adrian Schwarzer et al.   MOLECULAR THERAPY

Retrieval of vector integration sites from cell-free DNA

(2021) Daniela Cesana et al.   NATURE MEDICINE

Challenges in estimating numbers of vectors integrated in gene-modified cells using DNA sequence information

(2021) Frederic D. Bushman et al.   MOLECULAR THERAPY

Betibeglogene Autotemcel Gene Therapy for Non–β0/β0 Genotype β-Thalassemia

(2021) Franco Locatelli et al.   NEW ENGLAND JOURNAL OF MEDICINE

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A

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

Retroviral Insertional Mutagenesis in Humans: Evidence for Four Genetic Mechanisms Promoting Expansion of Cell Clones

(2020) Frederic D. Bushman   MOLECULAR THERAPY

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

(2020) Helena Costa Verdera et al.   MOLECULAR THERAPY

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

A Novel Next-Generation Sequencing and Analysis Platform to Assess the Identity of Recombinant Adeno-Associated Viral Preparations from Viral DNA Extracts

(2020) Karen Guerin et al.   HUMAN GENE THERAPY

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B

(2020) Lindsey A. George et al.   MOLECULAR THERAPY

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

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

Development of AAV Variants with Human Hepatocyte Tropism and Neutralizing Antibody Escape Capacity

(2020) Xiaolei Pei et al.   Molecular Therapy-Methods & Clinical Development

Current Clinical Applications of In Vivo Gene Therapy with AAVs

(2020) Jerry R. Mendell et al.   MOLECULAR THERAPY

Safety questions for AAV gene therapy

(2020) Charles P. Venditti   NATURE BIOTECHNOLOGY

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

Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges

(2019) Marina Cavazzana et al.   NATURE REVIEWS DRUG DISCOVERY

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy

(2019) Rithu Pattali et al.   GENE THERAPY

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

(2019) Tiziana La Bella et al.   GUT

Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease

(2019) Hye-Ri Kang et al.   Molecular Therapy-Methods & Clinical Development

AAV vector-mediated in vivo reprogramming into pluripotency

(2018) Elena Senís et al.   Nature Communications

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

Considerations for Preclinical Safety Assessment of Adeno-Associated Virus Gene Therapy Products

(2018) Basel T. Assaf et al.   TOXICOLOGIC PATHOLOGY

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

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

AAV Infection: Protection from Cancer

(2017) Arun Srivastava et al.   HUMAN GENE THERAPY

Points to Consider in Designing and Conducting Juvenile Toxicology Studies

(2017) Norman N. Kim et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

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

Identification of liver-specific enhancer–promoter activity in the 3′ untranslated region of the wild-type AAV2 genome

(2017) Grant J Logan et al.   NATURE GENETICS

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Next-generation AAV vectors for clinical use: an ever-accelerating race

(2017) Jonas Weinmann et al.   VIRUS GENES

INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes

(2017) Eric Sherman et al.   Molecular Therapy-Methods & Clinical Development

INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions

(2017) Charles C. Berry et al.   Molecular Therapy-Methods & Clinical Development

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

Cellular transduction mechanisms of adeno-associated viral vectors

(2016) Garrett Edward Berry et al.   Current Opinion in Virology

Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?

(2015) Kenneth I. Berns et al.   HUMAN GENE THERAPY

Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

(2015) Salima Hacein-Bey Abina et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

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

Adeno-associated Vector Toxicity—To Be or Not to Be?

(2015) Hildegard Büning et al.   MOLECULAR THERAPY

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

Translational Data from Adeno-Associated Virus-Mediated Gene Therapy of Hemophilia B in Dogs

(2015) Timothy C. Nichols et al.   Human Gene Therapy Clinical Development

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

Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo

(2014) Daniela Cesana et al.   MOLECULAR THERAPY

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

(2014) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene Therapy for Wiskott-Aldrich Syndrome--Long-Term Efficacy and Genotoxicity

(2014) C. J. Braun et al.   Science Translational Medicine

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

(2013) V Gauttier et al.   GENE THERAPY

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

Quantitative visualization of DNA G-quadruplex structures in human cells

(2013) Giulia Biffi et al.   Nature Chemistry

A largely random AAV integration profile after LPLD gene therapy

(2013) Christine Kaeppel et al.   NATURE MEDICINE

Lentiviral vector–based insertional mutagenesis identifies genes associated with liver cancer

(2013) Marco Ranzani et al.   NATURE METHODS

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

(2013) A. Aiuti et al.   SCIENCE

Incorporation of the factor IX Padua mutation into FIX-Triple improves clotting activity in vitro and in vivo

(2013) Chung-Yang Kao et al.   THROMBOSIS AND HAEMOSTASIS

Identification of Rtl1, a Retrotransposon-Derived Imprinted Gene, as a Novel Driver of Hepatocarcinogenesis

(2013) Jesse D. Riordan et al.   PLoS Genetics

From Bench to Bedside: Preclinical Evaluation of a Self-Inactivating Gammaretroviral Vector for the Gene Therapy of X-linked Chronic Granulomatous Disease

(2013) Stefan Stein et al.   Human Gene Therapy Clinical Development

Estimating abundances of retroviral insertion sites from DNA fragment length data

(2012) Charles C. Berry et al.   BIOINFORMATICS

Epidemiology of Viral Hepatitis and Hepatocellular Carcinoma

(2012) Hashem B. El-Serag   GASTROENTEROLOGY

Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations

(2012) Daniela Cesana et al.   JOURNAL OF CLINICAL INVESTIGATION

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

(2012) Lucia E Rosas et al.   MOLECULAR THERAPY

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

(2012) Ali Nowrouzi et al.   MOLECULAR THERAPY

Transcriptome Comparison between Fetal and Adult Mouse Livers: Implications for Circadian Clock Mechanisms

(2012) Chengwei Li et al.   PLoS One

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

Stable Human FIX Expression After 0.9G Intrauterine Gene Transfer of Self-complementary Adeno-associated Viral Vector 5 and 8 in Macaques

(2011) Citra NZ Mattar 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

Ex vivo gene transfer and correction for cell-based therapies

(2011) Luigi Naldini   NATURE REVIEWS GENETICS

Assessing the potential for AAV vector genotoxicity in a murine model

(2010) H. Li et al.   BLOOD

Molybdenum cofactor deficiency: Mutations in GPHN, MOCS1, and MOCS2

(2010) Jochen Reiss et al.   HUMAN MUTATION

Differential Effects of DNA Double-Strand Break Repair Pathways on Single-Strand and Self-Complementary Adeno-Associated Virus Vector Genomes

(2010) M. P. Cataldi et al.   JOURNAL OF VIROLOGY

Efficacy and Safety of Long-term Prophylaxis in Severe Hemophilia A Dogs Following Liver Gene Therapy Using AAV Vectors

(2010) Denise E Sabatino et al.   MOLECULAR THERAPY

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

(2010) Stefan Stein et al.   NATURE MEDICINE

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

A Modified Sleeping Beauty Transposon System That Can Be Used to Model a Wide Variety of Human Cancers in Mice

(2009) A. J. Dupuy et al.   CANCER RESEARCH

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

(2009) Eugenio Montini et al.   JOURNAL OF CLINICAL INVESTIGATION

Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral Vectors

(2009) Ute Modlich et al.   MOLECULAR THERAPY

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

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

Multi-stage analysis of gene expression and transcription regulation in C57/B6 mouse liver development

(2008) Tingting Li et al.   GENOMICS

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Molecular Characterization of the Heparin-Dependent Transduction Domain on the Capsid of a Novel Adeno-Associated Virus Isolate, AAV(VR-942)

(2008) M. Schmidt et al.   JOURNAL OF VIROLOGY

Leukemia induction after a single retroviral vector insertion in Evi1 or Prdm16

(2008) U Modlich et al.   LEUKEMIA

Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors

(2008) Daniela Zychlinski et al.   MOLECULAR THERAPY