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

Polyploidy in liver development, homeostasis and disease

(2020) Romain Donne et al.   Nature Reviews Gastroenterology & Hepatology

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

(2019) Tiziana La Bella et al.   GUT

Biology and significance of alpha‐fetoprotein in hepatocellular carcinoma

(2019) Peter R. Galle et al.   LIVER INTERNATIONAL

Knockdown of PEBP4 inhibits human glioma cell growth and invasive potential via ERK1/2 signaling pathway

(2018) Ren-qiang Huang et al.   MOLECULAR CARCINOGENESIS

High levels of coagulation factors and venous thrombosis risk: strongest association for factor VIII and von Willebrand factor

(2018) I. M. Rietveld et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

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

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

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

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

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

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

Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas

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

PEBP4 promoted the growth and migration of cancer cells in pancreatic ductal adenocarcinoma

(2015) Dexiang Zhang et al.   TUMOR BIOLOGY

Implementing a Class of Permutation Tests: ThecoinPackage

(2015) Torsten Hothorn et al.   Journal of Statistical Software

Comparing DNA integration site clusters with scan statistics

(2014) Charles C. Berry et al.   BIOINFORMATICS

NGS library preparation may generate artifactual integration sites of AAV vectors

(2014) Benjamin Cogné et al.   NATURE MEDICINE

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

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

Potential of regenerative medicine techniques in canine hepatology

(2014) Baukje A. Schotanus et al.   VETERINARY QUARTERLY

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

(2013) Li Zhong et al.   HUMAN GENE THERAPY

A largely random AAV integration profile after LPLD gene therapy

(2013) Christine Kaeppel et al.   NATURE MEDICINE

RefSeq: an update on mammalian reference sequences

(2013) Kim D. Pruitt et al.   NUCLEIC ACIDS RESEARCH

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

Estimating abundances of retroviral insertion sites from DNA fragment length data

(2012) Charles C. Berry et al.   BIOINFORMATICS

Molecular pathogenesis of chronic lymphocytic leukemia

(2012) Gianluca Gaidano 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

Assessing the potential for AAV vector genotoxicity in a murine model

(2010) H. Li et al.   BLOOD

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

Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model

(2009) D. E. Sabatino et al.   BLOOD