Related references
Note: Only part of the references are listed.Transcutaneous ultrasound-mediated gene delivery into canine livers achieves therapeutic levels of factor VIII expression
Megan A. Manson et al.
BLOOD ADVANCES (2022)
Induced Liver Regeneration Enhances CRISPR/Cas9-Mediated Gene Repair in Tyrosinemia Type 1
Qing-Shuo Zhang et al.
HUMAN GENE THERAPY (2021)
Sequential Activation of Guide RNAs to Enable Successive CRISPR-Cas9 Activities
Ryan Clarke et al.
MOLECULAR CELL (2021)
Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3
Min Qiu et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2021)
Therapeutic liver repopulation by transient acetaminophen selection of gene-modified hepatocytes
Anne Vonada et al.
SCIENCE TRANSLATIONAL MEDICINE (2021)
In vitro expansion of cirrhosis derived liver epithelial cells with defined small molecules
Bin Li et al.
STEM CELL RESEARCH (2021)
AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer
Helena Costa Verdera et al.
MOLECULAR THERAPY (2020)
Engineering adeno-associated virus vectors for gene therapy
Chengwen Li et al.
NATURE REVIEWS GENETICS (2020)
CRISPR-Cas12a delivery by DNA-mediated bioresponsive editing for cholesterol regulation
Wujin Sun et al.
SCIENCE ADVANCES (2020)
AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria
Daelyn Y. Richards et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
Adeno-associated virus vector as a platform for gene therapy delivery
Dan Wang et al.
NATURE REVIEWS DRUG DISCOVERY (2019)
Ribozyme-mediated, multiplex CRISPR gene editing and CRISPR interference (CRISPRi) in rodent-infectious Plasmodium yoelii
Michael P. Walker et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2019)
CHOPCHOP v3: expanding the CRISPR web toolbox beyond genome editing
Kornel Labun et al.
NUCLEIC ACIDS RESEARCH (2019)
Single transcript unit CRISPR 2.0 systems for robust Cas9 and Cas12a mediated plant genome editing
Xu Tang et al.
PLANT BIOTECHNOLOGY JOURNAL (2019)
Snakemake-a scalable bioinformatics workflow engine (vol 28, pg 2520, 2012)
Johannes Koster et al.
BIOINFORMATICS (2018)
Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9
Kshitiz Singh et al.
MOLECULAR THERAPY (2018)
Empower multiplex cell and tissue-specific CRISPR-mediated gene manipulation with self-cleaving ribozymes and tRNA
Li Xu et al.
NUCLEIC ACIDS RESEARCH (2017)
The emerging biology of RNA post-transcriptional modifications
Sigrid Nachtergaele et al.
RNA BIOLOGY (2017)
Self-cleaving ribozymes enable the production of guide RNAs from unlimited choices of promoters for CRISPR/Cas9 mediated genome editing
Yubing He et al.
JOURNAL OF GENETICS AND GENOMICS (2017)
Acetaminophen (APAP) hepatotoxicity-Isn't it time for APAP to go away?
William M. Lee
JOURNAL OF HEPATOLOGY (2017)
Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes
Brett T. Staahl et al.
NATURE BIOTECHNOLOGY (2017)
Identification of liver-specific enhancer-promoter activity in the 3′ untranslated region of the wild-type AAV2 genome
Grant J. Logan et al.
NATURE GENETICS (2017)
In Vivo Delivery of CRISPR/Cas9 for Therapeutic Gene Editing: Progress and Challenges
Rubul Mout et al.
BIOCONJUGATE CHEMISTRY (2017)
Guidelines for the optimal design of miRNA-based shRNAs
Xavier Bofill-De Ros et al.
METHODS (2016)
A Single Transcript CRISPR-Cas9 System for Efficient Genome Editing in Plants
Xu Tang et al.
MOLECULAR PLANT (2016)
Near-optimal probabilistic RNA-seq quantification
Nicolas L. Bray et al.
NATURE BIOTECHNOLOGY (2016)
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
Yang Yang et al.
NATURE BIOTECHNOLOGY (2016)
Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo
Hao Yin et al.
NATURE BIOTECHNOLOGY (2016)
Ribozyme Mediated gRNA Generation for In Vitro and In Vivo CRISPR/Cas9 Mutagenesis
Raymond Teck Ho Lee et al.
PLOS ONE (2016)
A universal system to select gene-modified hepatocytes in vivo
Sean Nygaard et al.
SCIENCE TRANSLATIONAL MEDICINE (2016)
Targeted Genome Editing via CRISPR in the Pathogen Cryptococcus neoformans
Samantha D. M. Arras et al.
PLOS ONE (2016)
Evaluation of off-target and on-target scoring algorithms and integration into the guide RNA selection tool CRISPOR
Maximilian Haeussler et al.
GENOME BIOLOGY (2016)
Cellular Homeostasis and Repair in the Mammalian Liver
Ben Z. Stanger
ANNUAL REVIEW OF PHYSIOLOGY, VOL 77 (2015)
miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction
Swaroopa Guda et al.
MOLECULAR THERAPY (2015)
CRISPR-Cas9-Mediated Genome Editing in Leishmania donovani
Wen-Wei Zhang et al.
MBIO (2015)
Development of a mono-promoter-driven CRISPR/Cas9 system in mammalian cells
Shin Yoshioka et al.
SCIENTIFIC REPORTS (2015)
Promoter less gene targeting without nucleases ameliorates haemophilia B in mice
A. Barzel et al.
NATURE (2015)
Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases
Sangsu Bae et al.
BIOINFORMATICS (2014)
CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling
Randall J. Platt et al.
CELL (2014)
Self- processing of ribozyme- flanked RNAs into guide RNAs in vitro and in vivo for CRISPR- mediated genome editing
Yangbin Gao et al.
JOURNAL OF INTEGRATIVE PLANT BIOLOGY (2014)
Multiplexed and Programmable Regulation of Gene Networks with an Integrated RNA and CRISPR/Cas Toolkit in Human Cells
Lior Nissim et al.
MOLECULAR CELL (2014)
Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates
Marinee K. Chuah et al.
MOLECULAR THERAPY (2014)
Easy quantitative assessment of genome editing by sequence trace decomposition
Eva K. Brinkman et al.
NUCLEIC ACIDS RESEARCH (2014)
An Optimized microRNA Backbone for Effective Single-Copy RNAi
Christof Fellmann et al.
CELL REPORTS (2013)
NADPH-cytochrome P450 oxidoreductase: Prototypic member of the diflavin reductase family
Takashi Iyanagi et al.
ARCHIVES OF BIOCHEMISTRY AND BIOPHYSICS (2012)
Snakemake-a scalable bioinformatics workflow engine
Johannes Koester et al.
BIOINFORMATICS (2012)
AAV-Mediated Gene Targeting Is Significantly Enhanced by Transient Inhibition of Nonhomologous End Joining or the Proteasome In Vivo
Nicole K. Paulk et al.
HUMAN GENE THERAPY (2012)
Polymerase II Promoter Strength Determines Efficacy of microRNA Adapted shRNAs
Robert Jan Lebbink et al.
PLOS ONE (2011)
A robust and high-throughput Cre reporting and characterization system for the whole mouse brain
Linda Madisen et al.
NATURE NEUROSCIENCE (2010)
Stem Cells and Liver Regeneration
Andrew W. Duncan et al.
GASTROENTEROLOGY (2009)
Ultrafast and memory-efficient alignment of short DNA sequences to the human genome
Ben Langmead et al.
GENOME BIOLOGY (2009)
Liver-specific deletion of the NADPH-cytochrome P450 reductase gene - Impact on plasma cholesterol homeostasis and the function and regulation of microsomal cytochrome P450 and heme oxygenase
J Gu et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2003)
An unusually compact external promoter for RNA polymerase III transcription of the human H1RNA gene
E Myslinski et al.
NUCLEIC ACIDS RESEARCH (2001)