A universal system to select gene-modified hepatocytes in vivo

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

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

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

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

Gene therapy enhances chemotherapy tolerance and efficacy in glioblastoma patients

(2014) Jennifer E. Adair et al.   JOURNAL OF CLINICAL INVESTIGATION

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

(2014) A. Barzel et al.   NATURE

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

Genetic Basis of Drug-Induced Liver Injury: Present and Future

(2014) Thomas Urban et al.   SEMINARS IN LIVER DISEASE

An Optimized microRNA Backbone for Effective Single-Copy RNAi

(2013) Christof Fellmann et al.   Cell Reports

AAV-Mediated Gene Targeting Is Significantly Enhanced by Transient Inhibition of Nonhomologous End Joining or the Proteasome In Vivo

(2012) Nicole K. Paulk et al.   HUMAN GENE THERAPY

Effect of nitisinone (NTBC) treatment on the clinical course of hepatorenal tyrosinemia in Québec

(2012) Jean Larochelle et al.   MOLECULAR GENETICS AND METABOLISM

In Vivo Selection of Transplanted Hepatocytes by Pharmacological Inhibition of Fumarylacetoacetate Hydrolase in Wild-type Mice

(2012) Nicole K Paulk et al.   MOLECULAR THERAPY

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

(2012) Zhongya Wang et al.   MOLECULAR THERAPY

Ribosomal DNA Integrating rAAV-rDNA Vectors Allow for Stable Transgene Expression

(2012) Leszek Lisowski 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

Metabolic activation in drug-induced liver injury

(2011) Louis Leung et al.   DRUG METABOLISM REVIEWS

Spontaneous hepatic repopulation in transgenic mice expressing mutant human α1-antitrypsin by wild-type donor hepatocytes

(2011) Jianqiang Ding et al.   JOURNAL OF CLINICAL INVESTIGATION

AAV8-mediated Hepatic Gene Transfer in Infant Rhesus Monkeys (Macaca mulatta)

(2011) Lili Wang et al.   MOLECULAR THERAPY

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

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

TBI during BM and SCT: review of the past, discussion of the present and consideration of future directions

(2010) C E Hill-Kayser et al.   BONE MARROW TRANSPLANTATION

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

(2009) Nicole K. Paulk et al.   HEPATOLOGY

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

(2009) Gary C. Pien et al.   JOURNAL OF CLINICAL INVESTIGATION