相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents
Xue Gao et al.
NATURE (2018)
Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy
Seuk-Min Ryu et al.
NATURE BIOTECHNOLOGY (2018)
Base editing with a Cpf1-cytidine deaminase fusion
Xiaosa Li et al.
NATURE BIOTECHNOLOGY (2018)
Programmable base editing of A.T to G.C in genomic DNA without DNA cleavage
Nicole M. Gaudelli et al.
NATURE (2017)
Correction of a pathogenic gene mutation in human embryos
Hong Ma et al.
NATURE (2017)
Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions
Y. Bill Kim et al.
NATURE BIOTECHNOLOGY (2017)
Engineered Cpf1 variants with altered PAM specificities
Linyi Gao et al.
NATURE BIOTECHNOLOGY (2017)
In Vivo Base Editing of PCSK9 (Proprotein Convertase Subtilisin/Kexin Type 9) as a Therapeutic Alternative to Genome Editing
Alexandra C. Chadwick et al.
ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY (2017)
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
Alexis C. Komor et al.
NATURE (2016)
CrispRVariants charts the mutation spectrum of genome engineering experiments
Helen Lindsay 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)
Teleseismic S wave microseisms
Kiwamu Nishida et al.
SCIENCE (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)
Crystal Structure of Staphylococcus aureus Cas9
Hiroshi Nishimasu et al.
CELL (2015)
Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System
Bernd Zetsche et al.
CELL (2015)
Engineered CRISPR-Cas9 nucleases with altered PAM specificities
Benjamin P. Kleinstiver et al.
NATURE (2015)
In vivo genome editing using Staphylococcus aureus Cas9
F. Ann Ran et al.
NATURE (2015)
Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition
Benjamin P. Kleinstiver et al.
NATURE BIOTECHNOLOGY (2015)
Development of an intein-mediated split-Cas9 system for gene therapy
Dong-Jiunn Jeffery Truong et al.
NUCLEIC ACIDS RESEARCH (2015)
PEAR: a fast and accurate Illumina Paired-End reAd mergeR
Jiajie Zhang et al.
BIOINFORMATICS (2014)
Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy
Nisha Nair et al.
BLOOD (2014)
Crystal Structure of Cas9 in Complex with Guide RNA and Target DNA
Hiroshi Nishimasu et al.
CELL (2014)
Treatment of Phenylketonuria Using Minicircle-Based Naked-DNA Gene Transfer to Murine Liver
Hiu Man Viecelli et al.
HEPATOLOGY (2014)
Phenotypic Reversion of Fair Hair upon Gene Therapy of the Phenylketonuria Mice
Beat Thoeny et al.
HUMAN GENE THERAPY (2014)
Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
Hao Yin et al.
NATURE BIOTECHNOLOGY (2014)
Redundant Sources of Wnt Regulate Intestinal Stem Cells and Promote Formation of Paneth Cells
Henner F. Farin et al.
GASTROENTEROLOGY (2012)
Quantification of phenylalanine hydroxylase activity by isotope-dilution liquid chromatography-electrospray ionization tandem mass spectrometry
Caroline Heintz et al.
MOLECULAR GENETICS AND METABOLISM (2012)
Phenylalanine hydroxylase deficiency
John J. Mitchell et al.
GENETICS IN MEDICINE (2011)
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
Federico Mingozzi et al.
NATURE REVIEWS GENETICS (2011)
State-of-the-art gene-based therapies: the road ahead
Mark A. Kay
NATURE REVIEWS GENETICS (2011)
Lgr5+ve Stem Cells Drive Self-Renewal in the Stomach and Build Long-Lived Gastric Units In Vitro
Nick Barker et al.
CELL STEM CELL (2010)
Animal models of brain dysfunction in phenylketonuria
A. E. Martynyuk et al.
MOLECULAR GENETICS AND METABOLISM (2010)
DNA repair by nonhomologous end joining and homologous recombination during cell cycle in human cells
Zhiyong Mao et al.
CELL CYCLE (2008)
Comparison of nonhomologous end joining and homologous recombination in human cells
Zhiyong Mao et al.
DNA REPAIR (2008)
Liver: An organ with predominant innate immunity
Bin Gao et al.
HEPATOLOGY (2008)
Epilepsy in phenylketonuria: A complex dependence on serum phenylalanine levels
Anatoly E. Martynyuk et al.
EPILEPSIA (2007)
Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer
Z Ding et al.
GENE THERAPY (2006)
Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria
CO Harding et al.
GENE THERAPY (2006)
The liver as an immunological organ
V Racanelli et al.
HEPATOLOGY (2006)
Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway
AM Davidoff et al.
BLOOD (2003)
分享论文
