In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice
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Gregory A. Newby | Luke W. Koblan | M. Erdos | N. Narisu | Christopher Wilson | Jonathan M. Levy | Q. Sheng | David R. Liu | Kan Cao | L. Gordon | Jonathan D. Brown | Xiaojing Mao | Zheng-Mei Xiong | Lindsay M Davison | S. P. Doherty | Chad Krilow | Charles Y. Lin | W. A. Cabral | Francis S. Collins | Yantenew G. Gete | Urraca L. Tavarez
[1] Wing‐Fu Lai,et al. Progress and trends in the development of therapies for Hutchinson–Gilford progeria syndrome , 2020, Aging cell.
[2] David R. Liu,et al. Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors , 2020, Nature Biotechnology.
[3] Jennifer A. Doudna,et al. Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity , 2020, Nature Biotechnology.
[4] Jonathan Yen,et al. Directed evolution of adenine base editors with increased activity and therapeutic application , 2020, Nature Biotechnology.
[5] David R. Liu,et al. Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses , 2019, Nature Biomedical Engineering.
[6] P. Magistretti,et al. Precise in vivo genome editing via single homology arm donor mediated intron-targeting gene integration for genetic disease correction , 2019, Cell Research.
[7] Tony P. Huang,et al. Circularly permuted and PAM-modified Cas9 variants broaden the targeting scope of base editors , 2019, Nature Biotechnology.
[8] V. Quesada,et al. Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome , 2019, Nature Medicine.
[9] Galina A. Erikson,et al. Single-Dose CRISPR/Cas9 Therapy Extends Lifespan of Mice with Hutchinson-Gilford Progeria Syndrome , 2018, Nature Medicine.
[10] M. A. Odena,et al. Next-Generation Sequencing and Quantitative Proteomics of Hutchinson-Gilford progeria syndrome-derived cells point to a role of nucleotide metabolism in premature aging , 2018, PloS one.
[11] C. Cole,et al. The COSMIC Cancer Gene Census: describing genetic dysfunction across all human cancers , 2018, Nature Reviews Cancer.
[12] M. Robinson,et al. Treatment of a metabolic liver disease by in vivo genome base editing in adult mice , 2018, Nature Medicine.
[13] E. Levanon,et al. A-to-I RNA editing — immune protector and transcriptome diversifier , 2018, Nature Reviews Genetics.
[14] David R. Liu,et al. Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction , 2018, Nature Biotechnology.
[15] R. D'Agostino,et al. Cardiac Abnormalities in Patients With Hutchinson-Gilford Progeria Syndrome , 2018, JAMA cardiology.
[16] Nicole M. Gaudelli,et al. Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage , 2017, Nature.
[17] J. Doudna,et al. CRISPR-Cas9 Structures and Mechanisms. , 2017, Annual review of biophysics.
[18] J. Joung,et al. CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR-Cas9 nuclease off-targets , 2017, Nature Methods.
[19] R. Chandler,et al. Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models. , 2017, Human gene therapy.
[20] C. López-Otín,et al. Cardiac electrical defects in progeroid mice and Hutchinson–Gilford progeria syndrome patients with nuclear lamina alterations , 2016, Proceedings of the National Academy of Sciences.
[21] Måns Magnusson,et al. MultiQC: summarize analysis results for multiple tools and samples in a single report , 2016, Bioinform..
[22] Jingang Liu,et al. Overexpression of Rab25 promotes hepatocellular carcinoma cell proliferation and invasion , 2016, Tumor Biology.
[23] J. Keith Joung,et al. 731. High-Fidelity CRISPR-Cas9 Nucleases with No Detectable Genome-Wide Off-Target Effects , 2016 .
[24] David R. Liu,et al. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage , 2016, Nature.
[25] Meagan E. Sullender,et al. Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9 , 2015, Nature Biotechnology.
[26] Edwin Cuppen,et al. Sambamba: fast processing of NGS alignment formats , 2015, Bioinform..
[27] Michael Recht,et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. , 2014, The New England journal of medicine.
[28] R. D'Agostino,et al. Impact of Farnesylation Inhibitors on Survival in Hutchinson-Gilford Progeria Syndrome , 2014, Circulation.
[29] Neville E. Sanjana,et al. Improved vectors and genome-wide libraries for CRISPR screening , 2014, Nature Methods.
[30] Howard Y. Chang,et al. Transposition of native chromatin for fast and sensitive epigenomic profiling of open chromatin, DNA-binding proteins and nucleosome position , 2013, Nature Methods.
[31] Mauricio O. Carneiro,et al. From FastQ Data to High‐Confidence Variant Calls: The Genome Analysis Toolkit Best Practices Pipeline , 2013, Current protocols in bioinformatics.
[32] David R. Liu,et al. High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity , 2013, Nature Biotechnology.
[33] F. Collins,et al. Use of microarray hybrid capture and next-generation sequencing to identify the anatomy of a transgene , 2013, Nucleic acids research.
[34] A. Giobbie-Hurder,et al. Clinical trial of a farnesyltransferase inhibitor in children with Hutchinson–Gilford progeria syndrome , 2012, Proceedings of the National Academy of Sciences.
[35] G. Smyth,et al. Camera: a competitive gene set test accounting for inter-gene correlation , 2012, Nucleic acids research.
[36] Lili Wang,et al. Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector. , 2012, Human gene therapy.
[37] A. Giobbie-Hurder,et al. Mechanisms of Premature Vascular Aging in Children With Hutchinson-Gilford Progeria Syndrome , 2012, Hypertension.
[38] I. Varela,et al. Splicing-Directed Therapy in a New Mouse Model of Human Accelerated Aging , 2011, Science Translational Medicine.
[39] Francis S Collins,et al. Rapamycin Reverses Cellular Phenotypes and Enhances Mutant Protein Clearance in Hutchinson-Gilford Progeria Syndrome Cells , 2011, Science Translational Medicine.
[40] M. DePristo,et al. A framework for variation discovery and genotyping using next-generation DNA sequencing data , 2011, Nature Genetics.
[41] F. Collins,et al. Cardiovascular Pathology in Hutchinson-Gilford Progeria: Correlation With the Vascular Pathology of Aging , 2010, Arteriosclerosis, thrombosis, and vascular biology.
[42] M. DePristo,et al. The Genome Analysis Toolkit: a MapReduce framework for analyzing next-generation DNA sequencing data. , 2010, Genome research.
[43] H. Hakonarson,et al. ANNOVAR: functional annotation of genetic variants from high-throughput sequencing data , 2010, Nucleic acids research.
[44] Aaron R. Quinlan,et al. BIOINFORMATICS APPLICATIONS NOTE , 2022 .
[45] Gonçalo R. Abecasis,et al. The Sequence Alignment/Map format and SAMtools , 2009, Bioinform..
[46] Richard Durbin,et al. Sequence analysis Fast and accurate short read alignment with Burrows – Wheeler transform , 2009 .
[47] J. Rabinowitz,et al. Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. , 2008, Molecular therapy : the journal of the American Society of Gene Therapy.
[48] D. Duan,et al. Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration , 2007, Gene Therapy.
[49] Daniel G. Miller,et al. AAV Vector Integration Sites in Mouse Hepatocellular Carcinoma , 2007, Science.
[50] Francis S. Collins,et al. Human laminopathies: nuclei gone genetically awry , 2006, Nature Reviews Genetics.
[51] F. Collins,et al. Progressive vascular smooth muscle cell defects in a mouse model of Hutchinson-Gilford progeria syndrome. , 2006, Proceedings of the National Academy of Sciences of the United States of America.
[52] Theresa A. Storm,et al. Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.
[53] T. Misteli,et al. Reversal of the cellular phenotype in the premature aging disease Hutchinson-Gilford progeria syndrome , 2005, Nature Medicine.
[54] Pierre Cau,et al. Lamin A Truncation in Hutchinson-Gilford Progeria , 2003, Science.
[55] Laura Scott,et al. Recurrent de novo point mutations in lamin A cause Hutchinson–Gilford progeria syndrome , 2003, Nature.
[56] T. Flotte,et al. Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors , 2001, Gene Therapy.
[57] P. D. de Jong,et al. A modular, positive selection bacterial artificial chromosome vector with multiple cloning sites. , 1999, Genomics.
[58] B. Byrne,et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. , 1996, Proceedings of the National Academy of Sciences of the United States of America.
[59] S. Imbeaud,et al. Wild-type AAV Insertions in Hepatocellular Carcinoma Do Not Inform Debate Over Genotoxicity Risk of Vectorized AAV. , 2016, Molecular therapy : the journal of the American Society of Gene Therapy.
[60] M. Sands. AAV-mediated liver-directed gene therapy. , 2011, Methods in molecular biology.
[61] T. Flotte,et al. 216. Long Term Portal Vein Administration of AAV-WPRE Vector Results in Increased Incidence of Neoplastic Disease and Hepatic Pathology , 2006 .
[62] E. Gilbert-Barness,et al. Histological and ultrastructural features of atherosclerosis in progeria. , 1999, Cardiovascular pathology : the official journal of the Society for Cardiovascular Pathology.