Liver-targeted gene therapy: Approaches and challenges

Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial

(2015) Paul E. Monahan et al.   HUMAN GENE THERAPY

Lysosomal storage disease: Gene therapy on both sides of the blood–brain barrier

(2015) Elena L. Aronovich et al.   MOLECULAR GENETICS AND METABOLISM

TALEN-mediated genome editing: prospects and perspectives

(2014) David A. Wright et al.   BIOCHEMICAL JOURNAL

Hepatocyte-targeting gene delivery using a lipoplex composed of galactose-modified aromatic lipid synthesized with click chemistry

(2014) Mizuha Sakashita et al.   BIOORGANIC & MEDICINAL CHEMISTRY

CRISPR/Cas9-mediated genome engineering: An adeno-associated viral (AAV) vector toolbox

(2014) Elena Senís et al.   Biotechnology Journal

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling

(2014) Randall J. Platt et al.   CELL

Gene Replacement Therapy for Genetic Hepatocellular Jaundice

(2014) Remco van Dijk et al.   CLINICAL REVIEWS IN ALLERGY & IMMUNOLOGY

Current status of gene therapy for α-1 antitrypsin deficiency

(2014) Heather S Loring et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

High and prolonged sulfamidase secretion by the liver of MPS-IIIA mice following hydrodynamic tail vein delivery of antibiotic-free pFAR4 plasmid vector

(2014) M Quiviger et al.   GENE THERAPY

Adenoviral transfer of human aquaporin-1 gene to rat liver improves bile flow in estrogen-induced cholestasis

(2014) J Marrone et al.   GENE THERAPY

Gene therapy for rhesus monkeys heterozygous for LDL receptor deficiency by balloon catheter hepatic delivery of helper-dependent adenoviral vector

(2014) K Oka et al.   GENE THERAPY

Off-target assessment of CRISPR-Cas9 guiding RNAs in human iPS and mouse ES cells

(2014) E-Pien Tan et al.   GENESIS

Current status of haemophilia gene therapy

(2014) K. H. High et al.   HAEMOPHILIA

Selective In Vivo Targeting of Human Liver Tumors by Optimized AAV3 Vectors in a Murine Xenograft Model

(2014) Chen Ling et al.   HUMAN GENE THERAPY

Hepatitis C Virus Entry Is Impaired by Claudin-1 Downregulation in Diacylglycerol Acyltransferase-1-Deficient Cells

(2014) P. S. Sung et al.   JOURNAL OF VIROLOGY

Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

(2014) Marinee K Chuah et al.   MOLECULAR THERAPY

Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy

(2014) Mario Di Matteo et al.   MOLECULAR THERAPY

CRISPR-mediated direct mutation of cancer genes in the mouse liver

(2014) Wen Xue et al.   NATURE

DNA interrogation by the CRISPR RNA-guided endonuclease Cas9

(2014) Samuel H. Sternberg et al.   NATURE

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

Engineering adeno-associated viruses for clinical gene therapy

(2014) Melissa A. Kotterman et al.   NATURE REVIEWS GENETICS

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennaryN-acetyl galactosamine improves potency 10-fold in mice

(2014) Thazha P. Prakash et al.   NUCLEIC ACIDS RESEARCH

Safety Assessment of Liver-Targeted Hydrodynamic Gene Delivery in Dogs

(2014) Kenya Kamimura et al.   PLoS One

Generation of Ugt1-Deficient Murine Liver Cell Lines Using TALEN Technology

(2014) Fabiola Porro et al.   PLoS One

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

Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates

(2014) Y. Dong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Small RNA- and DNA-based gene therapy for the treatment of liver cirrhosis, where we are?

(2014) Kyung-Hyun Kim   WORLD JOURNAL OF GASTROENTEROLOGY

Microhomology-mediated end-joining-dependent integration of donor DNA in cells and animals using TALENs and CRISPR/Cas9

(2014) Shota Nakade et al.   Nature Communications

Novel Adeno-associated Viruses Derived From Pig Tissues Transduce Most Major Organs in Mice

(2014) Alexander Bello et al.   Scientific Reports

Current Progress in Gene Delivery Technology Based on Chemical Methods and Nano-carriers

(2014) Lian Jin et al.   Theranostics

Correction of Hyperbilirubinemia in Gunn Rats by Surgical Delivery of Low Doses of Helper-Dependent Adenoviral Vectors

(2014) Françoise Schmitt et al.   Human Gene Therapy Methods

Mechanism of Polyplex- and Lipoplex-Mediated Delivery of Nucleic Acids: Real-Time Visualization of Transient Membrane Destabilization without Endosomal Lysis

(2013) Zia ur Rehman et al.   ACS Nano

Are human endogenous retroviruses pathogenic? An approach to testing the hypothesis

(2013) George R. Young et al.   BIOESSAYS

Transcription activator-like effector nucleases (TALENs): A highly efficient and versatile tool for genome editing

(2013) Ning Sun et al.   BIOTECHNOLOGY AND BIOENGINEERING

Robust ZFN-mediated genome editing in adult hemophilic mice

(2013) X. M. Anguela et al.   BLOOD

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

New gene delivery system based on oligochitosan and solid lipid nanoparticles: ‘In vitro’ and ‘in vivo’ evaluation

(2013) Diego Delgado et al.   EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

In the rat liver, Adenoviral gene transfer efficiency is comparable to AAV

(2013) P S Montenegro-Miranda et al.   GENE THERAPY

Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1

(2013) R J Chandler et al.   GENE THERAPY

Polyinosinic Acid Blocks Adeno-Associated Virus Macrophage Endocytosis In Vitro and Enhances Adeno-Associated Virus Liver-Directed Gene Therapy In Vivo

(2013) Remco van Dijk 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

Gene therapy and DNA delivery systems

(2013) D. Ibraheem et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

Rewriting a genome

(2013) Emmanuelle Charpentier et al.   NATURE

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

(2013) Leszek Lisowski et al.   NATURE

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity

(2013) Vikram Pattanayak et al.   NATURE BIOTECHNOLOGY

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Cas9 as a versatile tool for engineering biology

(2013) Prashant Mali et al.   NATURE METHODS

Anti-Tumor Activity of a miR-199-dependent Oncolytic Adenovirus

(2013) Elisa Callegari et al.   PLoS One

Adeno-Associated Viral Vector Serotype 5 Poorly Transduces Liver in Rat Models

(2013) Paula S. Montenegro-Miranda et al.   PLoS One

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

p12 Tethers the Murine Leukemia Virus Pre-integration Complex to Mitotic Chromosomes

(2013) Efrat Elis et al.   PLoS Pathogens

Sustained Knockdown of a Disease-Causing Gene in Patient-Specific Induced Pluripotent Stem Cells Using Lentiviral Vector-Based Gene Therapy

(2013) Reto Eggenschwiler et al.   Stem Cells Translational Medicine

Parameters Affecting Image-guided, Hydrodynamic Gene Delivery to Swine Liver

(2013) Kenya Kamimura et al.   Molecular Therapy-Nucleic Acids

Biodistribution of AAV8 Vectors Expressing Human Low-Density Lipoprotein Receptor in a Mouse Model of Homozygous Familial Hypercholesterolemia

(2013) Shu-Jen Chen et al.   Human Gene Therapy Clinical Development

Intrapleural Administration of an AAVrh.10 Vector Coding for Human α1-Antitrypsin for the Treatment of α1-Antitrypsin Deficiency

(2013) Maria J. Chiuchiolo et al.   Human Gene Therapy Clinical Development

A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR

(2012) H. Torikai et al.   BLOOD

Advances in Liver-Directed Gene Therapy for Hepatocellular Carcinoma by Non-Viral Delivery Systems

(2012) Buyun Ding et al.   CURRENT GENE THERAPY

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

(2012) D Gaudet et al.   GENE THERAPY

Therapeutic potential of RNA interference: a new molecular approach to antiviral treatment for hepatitis C

(2012) M. Motavaf et al.   JOURNAL OF VIRAL HEPATITIS

Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles

(2012) Christian Mueller et al.   MOLECULAR THERAPY

RNA-guided genetic silencing systems in bacteria and archaea

(2012) Blake Wiedenheft et al.   NATURE

Long-term, efficient inhibition of microRNA function in mice using rAAV vectors

(2012) Jun Xie et al.   NATURE METHODS

A transcription activator-like effector toolbox for genome engineering

(2012) Neville E Sanjana et al.   Nature Protocols

Inhibition of Hepatitis B Virus and Induction of Hepatoma Cell Apoptosis by ASGPR-Directed Delivery of shRNAs

(2012) Jingwei Ma et al.   PLoS One

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

A Wolf in Sheep's Clothing: SV40 Co-opts Host Genome Maintenance Proteins to Replicate Viral DNA

(2012) Gregory A. Sowd et al.   PLoS Pathogens

A Hyperactive Transposase Promotes Persistent Gene Transfer of a piggyBac DNA Transposon

(2012) Erin R Burnight et al.   Molecular Therapy-Nucleic Acids

CRISPR-Cas Systems in Bacteria and Archaea: Versatile Small RNAs for Adaptive Defense and Regulation

(2011) Devaki Bhaya et al.   Annual Review of Genetics

A gene delivery system for human cells mediated by both a cell-penetrating peptide and a piggyBac transposase

(2011) Cheng-Yi Lee et al.   BIOMATERIALS

The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver

(2011) A. T. Martino et al.   BLOOD

DNA triplex-mediated inhibition of MET leads to cell death and tumor regression in hepatoma

(2011) G Singhal et al.   CANCER GENE THERAPY

Retroviral Vector-mediated Gene Therapy for Metabolic Diseases: An Update

(2011) Nicolas Ferry et al.   CURRENT PHARMACEUTICAL DESIGN

Long-term phenotypic correction in factor IX knockout mice by using phiC31 integrase-mediated gene therapy

(2011) A Keravala et al.   GENE THERAPY

DNA delivery to ‘ex vivo’ human liver segments

(2011) M J Herrero et al.   GENE THERAPY

Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk

(2011) Janka Mátrai et al.   HEPATOLOGY

Rapid generation of mature hepatocyte-like cells from human induced pluripotent stem cells by an efficient three-step protocol

(2011) Yu-Fan Chen et al.   HEPATOLOGY

Infusing CD19-Directed T Cells to Augment Disease Control in Patients Undergoing Autologous Hematopoietic Stem-Cell Transplantation for Advanced B-Lymphoid Malignancies

(2011) Partow Kebriaei et al.   HUMAN GENE THERAPY

Hyperactive piggyBac Gene Transfer in Human Cells and In Vivo

(2011) Joseph E. Doherty et al.   HUMAN GENE THERAPY

Long-Term Expression of Human Coagulation Factor VIII in a Tolerant Mouse Model Using the φC31 Integrase System

(2011) Christopher L. Chavez et al.   HUMAN GENE THERAPY

The Sleeping Beauty transposon system: a non-viral vector for gene therapy

(2011) E. L. Aronovich et al.   HUMAN MOLECULAR GENETICS

Targeted TFO delivery to hepatic stellate cells

(2011) Ningning Yang et al.   JOURNAL OF CONTROLLED RELEASE

Gene therapy for haemophilia: a long and winding road

(2011) K. A. HIGH   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

HCCS1-armed, quadruple-regulated oncolytic adenovirus specific for liver cancer as a cancer targeting gene-viro-therapy strategy

(2011) Hai-Neng Xu et al.   Molecular Cancer

Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression

(2011) Jennifer B Nietupski et al.   MOLECULAR THERAPY

Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

(2011) Kosuke Yusa et al.   NATURE

CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III

(2011) Elitza Deltcheva et al.   NATURE

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

(2011) Hojun Li et al.   NATURE

Genetic engineering of human pluripotent cells using TALE nucleases

(2011) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Harnessing the potential of induced pluripotent stem cells for regenerative medicine

(2011) Sean M. Wu et al.   NATURE CELL BIOLOGY

RNAi-based therapeutic strategies for metabolic disease

(2011) Michael P. Czech et al.   Nature Reviews Endocrinology

State-of-the-art gene-based therapies: the road ahead

(2011) Mark A. Kay   NATURE REVIEWS GENETICS

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes

(2011) Erich Koller et al.   NUCLEIC ACIDS RESEARCH

Immune Activation and Target Organ Damage Are Consequences of Hydrodynamic Treatment but Not Delivery of Naked siRNAs in Mice

(2011) Zsuzsanna Rácz et al.   Nucleic Acid Therapeutics

Liver-targeted siRNA delivery by polyethylenimine (PEI)-pullulan carrier

(2010) Jeong-Hun Kang et al.   BIOORGANIC & MEDICINAL CHEMISTRY

Therapeutic applications of ribozymes and riboswitches

(2010) Jérôme Mulhbacher et al.   CURRENT OPINION IN PHARMACOLOGY

Heme oxygenase-1 gene delivery by Sleeping Beauty inhibits vascular stasis in a murine model of sickle cell disease

(2010) John D. Belcher et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

piggyBac Transposon-mediated Long-term Gene Expression in Mice

(2010) Hideyuki Nakanishi 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

The CRISPR/Cas bacterial immune system cleaves bacteriophage and plasmid DNA

(2010) Josiane E. Garneau et al.   NATURE

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

CRISPR/Cas, the Immune System of Bacteria and Archaea

(2010) P. Horvath et al.   SCIENCE

In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance

(2009) A. Annoni et al.   BLOOD

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

(2009) Nicole K. Paulk et al.   HEPATOLOGY

Highly efficient generation of human hepatocyte-like cells from induced pluripotent stem cells

(2009) Karim Si-Tayeb et al.   HEPATOLOGY

PiggyBac Transposon-based Inducible Gene Expression In Vivo After Somatic Cell Gene Transfer

(2009) Sai K Saridey et al.   MOLECULAR THERAPY

A Simple Cipher Governs DNA Recognition by TAL Effectors

(2009) M. J. Moscou et al.   SCIENCE

PiggyBac Transposon-Mediated, Reversible Gene Transfer in Human Embryonic Stem Cells

(2009) You-Tzung Chen et al.   STEM CELLS AND DEVELOPMENT

Redirecting Specificity of T-Cell Populations For CD19 Using the Sleeping Beauty System

(2008) H. Singh et al.   CANCER RESEARCH

Critical Parameters for Genome Editing Using Zinc Finger Nucleases

(2008) Todd Camenisch et al.   MINI-REVIEWS IN MEDICINAL CHEMISTRY

Sleeping Beauty Vector System Moves Toward Human Trials in the United States

(2008) David A Williams   MOLECULAR THERAPY

Development of a single-chain, quasi-dimeric zinc-finger nuclease for the selective degradation of mutated human mitochondrial DNA

(2008) Michal Minczuk et al.   NUCLEIC ACIDS RESEARCH

Chromosomal transposition of PiggyBac in mouse embryonic stem cells

(2008) W. Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA