Genome Editing Redefines Precision Medicine in the Cardiovascular Field
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Markus Krane | Elda Dzilic | Harald Lahm | Martina Dreßen | Marcus-André Deutsch | Rüdiger Lange | Sean M Wu | Stefanie A Doppler | R. Lange | M. Deutsch | M. Krane | Sean M. Wu | H. Lahm | S. Doppler | M. Dreßen | E. Dzilic
[1] Nicole M. Gaudelli,et al. Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage , 2017, Nature.
[2] Max A. Horlbeck,et al. Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation , 2014, Cell.
[3] Ying Sun,et al. CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes , 2015, Protein & Cell.
[4] Y. Doyon,et al. Targeted gene addition to a predetermined site in the human genome using a ZFN-based nicking enzyme , 2012, Genome research.
[5] George Church,et al. Titin mutations in iPS cells define sarcomere insufficiency as a cause of dilated cardiomyopathy , 2015, Science.
[6] Mengmeng Gong,et al. CRISPR/Cas9-mediated gene editing in human zygotes using Cas9 protein , 2017, Molecular Genetics and Genomics.
[7] M. Ohkura,et al. Allogeneic transplantation of iPS cell-derived cardiomyocytes regenerates primate hearts , 2016, Nature.
[8] Lei Wang,et al. Generation of Gene-Modified Cynomolgus Monkey via Cas9/RNA-Mediated Gene Targeting in One-Cell Embryos , 2014, Cell.
[9] M. Hasegawa,et al. Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome , 2009, Proceedings of the Japan Academy. Series B, Physical and biological sciences.
[10] G. Church,et al. Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription. , 2011, Nature biotechnology.
[11] Robert L Wilensky,et al. A quantitative, randomized study evaluating three methods of mesenchymal stem cell delivery following myocardial infarction. , 2006, European heart journal.
[12] Sean M. Wu,et al. Untangling the Biology of Genetic Cardiomyopathies with Pluripotent Stem Cell Disease Models , 2017, Current Cardiology Reports.
[13] C. Mummery,et al. Subtype-specific promoter-driven action potential imaging for precise disease modelling and drug testing in hiPSC-derived cardiomyocytes. , 2016, European heart journal.
[14] E. Tartour,et al. Human embryonic stem cell-derived cardiac progenitors for severe heart failure treatment: first clinical case report. , 2015, European heart journal.
[15] M. Mann,et al. Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy , 2015, EMBO molecular medicine.
[16] Hui Zhao,et al. Direct activation of human and mouse Oct4 genes using engineered TALE and Cas9 transcription factors , 2014, Nucleic acids research.
[17] Kun Zhang,et al. Whole-genome sequencing analysis reveals high specificity of CRISPR/Cas9 and TALEN-based genome editing in human iPSCs. , 2014, Cell stem cell.
[18] C. Mummery,et al. Subtype-specific promoter-driven action potential imaging for precise disease modelling and drug testing in hiPSC-derived cardiomyocytes , 2016, European heart journal.
[19] N. Benvenisty,et al. Quality control Genome maintenance in pluripotent stem cells , 2014 .
[20] George A. Truskey,et al. Modeling the mitochondrial cardiomyopathy of Barth syndrome with iPSC and heart-on-chip technologies , 2014, Nature Medicine.
[21] L. García,et al. Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice , 2013, EMBO molecular medicine.
[22] K. Pollard,et al. Human Disease Modeling Reveals Integrated Transcriptional and Epigenetic Mechanisms of NOTCH1 Haploinsufficiency , 2015, Cell.
[23] J. Keith Joung,et al. Improving CRISPR-Cas nuclease specificity using truncated guide RNAs , 2014, Nature Biotechnology.
[24] M. Horie,et al. Allele‐specific ablation rescues electrophysiological abnormalities in a human iPS cell model of long‐QT syndrome with a CALM2 mutation , 2017, Human molecular genetics.
[25] Ronald A. Li,et al. Correction of human phospholamban R14del mutation associated with cardiomyopathy using targeted nucleases and combination therapy , 2015, Nature Communications.
[26] J. Keith Joung,et al. High frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells , 2013, Nature Biotechnology.
[27] Jacob E Corn,et al. Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA , 2016, Nature Biotechnology.
[28] Martin J. Aryee,et al. GUIDE-Seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases , 2014, Nature Biotechnology.
[29] Luke A. Gilbert,et al. Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression , 2013, Cell.
[30] David A. Brafman,et al. May I Cut in? Gene Editing Approaches in Human Induced Pluripotent Stem Cells , 2017, Cells.
[31] Kevin T. Zhao,et al. Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions , 2017, Nature Biotechnology.
[32] Charles E. Murry,et al. Human Embryonic Stem Cell-Derived Cardiomyocytes Regenerate Non-Human Primate Hearts , 2014, Nature.
[33] T. Doetschman,et al. muscular dystrophies Gene Editing With CRISPR / Cas 9 RNA-Directed Nuclease , 2017 .
[34] Simona Casini,et al. Isogenic human pluripotent stem cell pairs reveal the role of a KCNH2 mutation in long-QT syndrome , 2013, The EMBO journal.
[35] A. Hendel,et al. A Comprehensive TALEN-Based Knockout Library for Generating Human-Induced Pluripotent Stem Cell–Based Models for Cardiovascular Diseases , 2017, Circulation research.
[36] M. Kay,et al. Genome editing of isogenic human induced pluripotent stem cells recapitulates long QT phenotype for drug testing. , 2014, Journal of the American College of Cardiology.
[37] David A. Scott,et al. Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity , 2013, Cell.
[38] International Human Genome Sequencing Consortium. Initial sequencing and analysis of the human genome , 2001, Nature.
[39] Steve D. M. Brown,et al. Correction of the auditory phenotype in C57BL/6N mice via CRISPR/Cas9-mediated homology directed repair , 2016, Genome Medicine.
[40] G. Fishman,et al. Identification and Purification of Human Induced Pluripotent Stem Cell-Derived Atrial-Like Cardiomyocytes Based on Sarcolipin Expression , 2014, PloS one.
[41] Martin J. Aryee,et al. Engineered CRISPR-Cas9 nucleases with altered PAM specificities , 2015, Nature.
[42] J. Joung,et al. Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition , 2015, Nature Biotechnology.
[43] B. Li,et al. Expression profiling reveals off-target gene regulation by RNAi , 2003, Nature Biotechnology.
[44] D. Malan,et al. Differential Expression Levels of Integrin α6 Enable the Selective Identification and Isolation of Atrial and Ventricular Cardiomyocytes , 2015, PloS one.
[45] Richard L. Frock,et al. Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases , 2014, Nature Biotechnology.
[46] Denise E. Waldron. Gene therapy: In vivo gene editing in non-dividing cells , 2017, Nature Reviews Genetics.
[47] George M. Church,et al. In vivo gene editing in dystrophic mouse muscle and muscle stem cells , 2016, Science.
[48] Desheng Liang,et al. TALE nickase mediates high efficient targeted transgene integration at the human multi-copy ribosomal DNA locus. , 2014, Biochemical and biophysical research communications.
[49] John M. Shelton,et al. Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy , 2016, Science.
[50] A. Haverich,et al. Transplantation of purified iPSC-derived cardiomyocytes in myocardial infarction , 2017, PloS one.
[51] Yarden Katz,et al. Multiplexed activation of endogenous genes by CRISPR-on, an RNA-guided transcriptional activator system , 2013, Cell Research.
[52] Terran Lane,et al. A computational study of off-target effects of RNA interference , 2005, Nucleic acids research.
[53] David R. Liu,et al. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage , 2016, Nature.
[54] K. Maher,et al. Molecular beacon-based detection and isolation of working-type cardiomyocytes derived from human pluripotent stem cells. , 2015, Biomaterials.
[55] J. Keith Joung,et al. 731. High-Fidelity CRISPR-Cas9 Nucleases with No Detectable Genome-Wide Off-Target Effects , 2016 .
[56] Sara Mantero,et al. Clinical transplantation of a tissue-engineered airway , 2008, The Lancet.
[57] Edward M. Callaway,et al. In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration , 2016, Nature.
[58] J. C. Tsang,et al. Reprogramming to Pluripotency Using Designer TALE Transcription Factors Targeting Enhancers , 2013, Stem cell reports.
[59] Michael P Snyder,et al. iPSC-derived cardiomyocytes reveal abnormal TGFβ signaling in left ventricular non-compaction cardiomyopathy , 2016, Nature Cell Biology.
[60] S. Yamanaka,et al. Induction of Pluripotent Stem Cells from Mouse Embryonic and Adult Fibroblast Cultures by Defined Factors , 2006, Cell.
[61] Hans Clevers,et al. Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients. , 2013, Cell stem cell.
[62] T. Ichisaka,et al. Induction of Pluripotent Stem Cells from Adult Human Fibroblasts by Defined Factors , 2007, Cell.
[63] Yuki Masumura,et al. Targeted Genome Replacement via Homology-directed Repair in Non-dividing Cardiomyocytes , 2017, Scientific Reports.
[64] C. Mason,et al. A brief definition of regenerative medicine. , 2008, Regenerative medicine.
[65] Jonathan C. Cohen,et al. Sequence variations in PCSK9, low LDL, and protection against coronary heart disease. , 2006, The New England journal of medicine.
[66] Chad A. Cowan,et al. Genome-Edited Human Pluripotent Stem Cell–Derived Macrophages as a Model of Reverse Cholesterol Transport—Brief Report , 2016, Arteriosclerosis, thrombosis, and vascular biology.
[67] Johan Verjans,et al. Myocardial remodeling after infarction: the role of myofibroblasts , 2010, Nature Reviews Cardiology.
[68] R. Ramirez,et al. Myosin light chain 2-based selection of human iPSC-derived early ventricular cardiac myocytes. , 2013, Stem cell research.
[69] ジョン,ジェー.キース,et al. Engineered CRISPR-Cas9 nucleases with altered PAM specificity , 2016 .
[70] Dongsheng Duan,et al. In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy , 2016, Science.
[71] G. Church,et al. CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering , 2013, Nature Biotechnology.
[72] Christopher M. Vockley,et al. RNA-guided gene activation by CRISPR-Cas9-based transcription factors , 2013, Nature Methods.
[73] C. Robertson,et al. Concise Review: Maturation Phases of Human Pluripotent Stem Cell‐Derived Cardiomyocytes , 2013, Stem cells.
[74] M. Hoehn,et al. Bioluminescent Imaging of Genetically Selected Induced Pluripotent Stem Cell-Derived Cardiomyocytes after Transplantation into Infarcted Heart of Syngeneic Recipients , 2014, PloS one.
[75] C. Antos,et al. Making human cardiomyocytes up to date: Derivation, maturation state and perspectives. , 2017, International journal of cardiology.
[76] Charles A. Gersbach,et al. A CRISPR/Cas9-Based System for Reprogramming Cell Lineage Specification , 2014, Stem cell reports.
[77] Rudolf Jaenisch,et al. One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering , 2013, Cell.
[78] Alain Bel,et al. Towards a clinical use of human embryonic stem cell-derived cardiac progenitors: a translational experience. , 2015, European heart journal.
[79] Jianhui Gong,et al. Correction of a pathogenic gene mutation in human embryos , 2018, Yearbook of Paediatric Endocrinology.
[80] Daniel J. Rader,et al. Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing , 2014, Circulation research.
[81] H. Leonhardt,et al. Targeted transcriptional activation of silent oct4 pluripotency gene by combining designer TALEs and inhibition of epigenetic modifiers , 2012, Nucleic acids research.
[82] Gang Bao,et al. CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity , 2013, Nucleic acids research.