Applications of Genome Editing Technology in Animal Disease Modeling and Gene Therapy

[1]  M. McCarthy Harnessing the potential of CRISPR-based platforms to advance the field of hospital medicine , 2020, Expert review of anti-infective therapy.

[2]  Charles A Gersbach,et al.  Increasing the specificity of CRISPR systems with engineered RNA secondary structures , 2019, Nature Biotechnology.

[3]  V. Quesada,et al.  Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome , 2019, Nature Medicine.

[4]  Galina A. Erikson,et al.  Single-Dose CRISPR/Cas9 Therapy Extends Lifespan of Mice with Hutchinson-Gilford Progeria Syndrome , 2018, Nature Medicine.

[5]  B. Beutler,et al.  A viable hypomorphic Arnt2 mutation causes hyperphagic obesity, diabetes and hepatic steatosis , 2018, Disease Models & Mechanisms.

[6]  Charles D. Yeh,et al.  Unbiased detection of CRISPR off-targets in vivo using DISCOVER-Seq , 2018, Science.

[7]  Petra Reinke,et al.  High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population , 2018, Nature Medicine.

[8]  John M. Shelton,et al.  Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy , 2018, Science.

[9]  W. Pavan,et al.  Modeling Niemann-Pick disease type C1 in zebrafish: a robust platform for in vivo screening of candidate therapeutic compounds , 2018, Disease Models & Mechanisms.

[10]  P. Zamore,et al.  Diseases caused by different mutations in the two alleles of a gene are treated in mice by Cas-9-induced allelic exchange. , 2018, Nature Biotechnology.

[11]  Martin J. Aryee,et al.  In vivo CRISPR editing with no detectable genome-wide off-target mutations , 2018, Nature.

[12]  D. Duan Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy , 2018, Molecular therapy : the journal of the American Society of Gene Therapy.

[13]  G. Stamatoyannopoulos,et al.  Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing. , 2018, Blood.

[14]  Gang Bao,et al.  A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human haematopoietic stem and progenitor cells , 2018, Nature Medicine.

[15]  L. Lai,et al.  A novel rabbit model of Duchenne muscular dystrophy generated by CRISPR/Cas9 , 2018, Disease Models & Mechanisms.

[16]  G. Chen,et al.  Exposing cancer with CRISPR-Cas9: from genetic identification to clinical therapy , 2018, Translational Cancer Research.

[17]  Adam Bagg,et al.  Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia , 2018, Cell.

[18]  Shihua Li,et al.  A Huntingtin Knockin Pig Model Recapitulates Features of Selective Neurodegeneration in Huntington’s Disease , 2018, Cell.

[19]  S. S. St Martin,et al.  Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing , 2018, Molecular therapy : the journal of the American Society of Gene Therapy.

[20]  E. Sorokina,et al.  PITX2 deficiency and associated human disease: insights from the zebrafish model , 2018, Human molecular genetics.

[21]  David R. Liu,et al.  Evolved Cas9 variants with broad PAM compatibility and high DNA specificity , 2018, Nature.

[22]  Maryam Clausen,et al.  Therapeutic Genome Editing With CRISPR/Cas9 in a Humanized Mouse Model Ameliorates α1-antitrypsin Deficiency Phenotype , 2018, EBioMedicine.

[23]  Ping Wang,et al.  In Vivo Ovarian Cancer Gene Therapy Using CRISPR-Cas9. , 2018, Human gene therapy.

[24]  Xuejin Chen,et al.  Production of Wilson Disease Model Rabbits with Homology-Directed Precision Point Mutations in the ATP7B Gene Using the CRISPR/Cas9 System , 2018, Scientific Reports.

[25]  B. An,et al.  NCKX3 was compensated by calcium transporting genes and bone resorption in a NCKX3 KO mouse model , 2017, Molecular and Cellular Endocrinology.

[26]  Luigi Naldini,et al.  Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1 , 2017, Science Translational Medicine.

[27]  Dennis Normile,et al.  China sprints ahead in CRISPR therapy race. , 2017, Science.

[28]  J. Bonkowsky,et al.  A zebrafish model of X-linked adrenoleukodystrophy recapitulates key disease features and demonstrates a developmental requirement for abcd1 in oligodendrocyte patterning and myelination , 2017, Human molecular genetics.

[29]  Jennifer A. Doudna,et al.  Enhanced proofreading governs CRISPR-Cas9 targeting accuracy , 2017, Nature.

[30]  Yuan He,et al.  CRISPR/Cas9-Mediated CCR5 Ablation in Human Hematopoietic Stem/Progenitor Cells Confers HIV-1 Resistance In Vivo. , 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

[31]  P. Tam,et al.  Correction of Hirschsprung-Associated Mutations in Human Induced Pluripotent Stem Cells Via Clustered Regularly Interspaced Short Palindromic Repeats/Cas9, Restores Neural Crest Cell Function. , 2017, Gastroenterology.

[32]  O. Nureki,et al.  CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice , 2017, Scientific Reports.

[33]  M. Malumbres,et al.  The anaphase promoting complex impacts repair choice by protecting ubiquitin signalling at DNA damage sites , 2017, Nature Communications.

[34]  Jin-Soo Kim,et al.  Selective disruption of an oncogenic mutant allele by CRISPR/Cas9 induces efficient tumor regression , 2017, Nucleic acids research.

[35]  Dinggang Shen,et al.  Modeling Rett Syndrome Using TALEN-Edited MECP2 Mutant Cynomolgus Monkeys , 2017, Cell.

[36]  Ke Men,et al.  Non-viral and viral delivery systems for CRISPR-Cas9 technology in the biomedical field , 2017, Science China Life Sciences.

[37]  Yang Yang,et al.  CRISPR/Cas9-mediated correction of human genetic disease , 2017, Science China Life Sciences.

[38]  Kui Li,et al.  CRISPR/Cas9-mediated ApoE-/- and LDLR-/- double gene knockout in pigs elevates serum LDL-C and TC levels , 2017, Oncotarget.

[39]  Claudio Mussolino,et al.  Refining strategies to translate genome editing to the clinic , 2017, Nature Medicine.

[40]  Y. Kamei,et al.  Protanopia (red color-blindness) in medaka: a simple system for producing color-blind fish and testing their spectral sensitivity , 2017, BMC Genetics.

[41]  Ning Wang,et al.  Artificial Virus Delivers CRISPR-Cas9 System for Genome Editing of Cells in Mice. , 2017, ACS nano.

[42]  Michael K. Wendt,et al.  The paradoxical functions of EGFR during breast cancer progression , 2017, Signal Transduction and Targeted Therapy.

[43]  A. Cheng,et al.  CRISPR-Cas9-mediated multiplex gene editing in CAR-T cells , 2016, Cell Research.

[44]  R. Sedláček,et al.  A viable mouse model for Netherton syndrome based on mosaic inactivation of the Spink5 gene , 2016, Biological chemistry.

[45]  A. Sahebkar,et al.  Molecular imaging and cancer gene therapy. , 2016, Cancer gene therapy.

[46]  Sruthi Mantri,et al.  CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells , 2016, Nature.

[47]  D. Deforce,et al.  CRISPR/Cas9 mediated knockout of rb1 and rbl1 leads to rapid and penetrant retinoblastoma development in Xenopus tropicalis , 2016, Scientific Reports.

[48]  Prashant Mali,et al.  A multifunctional AAV–CRISPR–Cas9 and its host response , 2016, Nature Methods.

[49]  J. Nesland,et al.  Generation of TALEN-mediated FH knockout rat model , 2016, Oncotarget.

[50]  Tetsushi Sakuma,et al.  Generation of a Nonhuman Primate Model of Severe Combined Immunodeficiency Using Highly Efficient Genome Editing. , 2016, Cell stem cell.

[51]  Ming-Yu Liu,et al.  Generation of obese rat model by transcription activator-like effector nucleases targeting the leptin receptor gene , 2016, Science China Life Sciences.

[52]  Y. Niu,et al.  Genome editing in nonhuman primates: approach to generating human disease models , 2016, Journal of internal medicine.

[53]  K. Horiuchi,et al.  Generation of heterozygous fibrillin-1 mutant cloned pigs from genome-edited foetal fibroblasts , 2016, Scientific Reports.

[54]  Christian Veltkamp,et al.  Multiplexed pancreatic genome engineering and cancer induction by transfection-based CRISPR/Cas9 delivery in mice , 2016, Nature Communications.

[55]  L. Lai,et al.  CRISPR/Cas9-mediated GJA8 knockout in rabbits recapitulates human congenital cataracts , 2016, Scientific Reports.

[56]  Yang Yang,et al.  A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice , 2016, Nature Biotechnology.

[57]  Daniel G. Anderson,et al.  Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo , 2016, Nature Biotechnology.

[58]  Ravi Karra,et al.  Single epicardial cell transcriptome sequencing identifies Caveolin 1 as an essential factor in zebrafish heart regeneration , 2016, Development.

[59]  L. Zentilin,et al.  A mouse model for adult cardiac-specific gene deletion with CRISPR/Cas9 , 2015, Proceedings of the National Academy of Sciences.

[60]  H. Harn,et al.  Targeting New Candidate Genes by Small Molecules Approaching Neurodegenerative Diseases , 2015, International journal of molecular sciences.

[61]  Margaret B. Fish,et al.  Xenopus pax6 mutants affect eye development and other organ systems, and have phenotypic similarities to human aniridia patients. , 2015, Developmental biology.

[62]  David V Schaffer,et al.  Viral Vectors for Gene Therapy: Translational and Clinical Outlook. , 2015, Annual review of biomedical engineering.

[63]  Meagan E. Sullender,et al.  Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9 , 2015, Nature Biotechnology.

[64]  Ning Li,et al.  Generation of a miniature pig disease model for human Laron syndrome , 2015, Scientific Reports.

[65]  C. Smythe,et al.  klf2a sh317 Mutant Zebrafish Do Not Recapitulate Morpholino-Induced Vascular and Haematopoietic Phenotypes , 2015, PloS one.

[66]  Rahul C. Deo,et al.  An internal promoter underlies the difference in disease severity between N- and C-terminal truncation mutations of Titin in zebrafish , 2015, eLife.

[67]  Y. Doyon,et al.  In vivo genome editing of the albumin locus as a platform for protein replacement therapy. , 2015, Blood.

[68]  A. Regev,et al.  Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System , 2015, Cell.

[69]  Jin-Soo Kim,et al.  Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9. , 2015, Cell stem cell.

[70]  Qiang Liu,et al.  Bioluminescent imaging of vaccinia virus infection in immunocompetent and immunodeficient rats as a model for human smallpox , 2015, Scientific Reports.

[71]  D. Stainier,et al.  Genetic compensation induced by deleterious mutations but not gene knockdowns , 2015, Nature.

[72]  De-Pei Liu,et al.  Both TALENs and CRISPR/Cas9 directly target the HBB IVS2–654 (C > T) mutation in β-thalassemia-derived iPSCs , 2015, Scientific Reports.

[73]  Volker Hovestadt,et al.  Somatic CRISPR/Cas9-mediated tumour suppressor disruption enables versatile brain tumour modelling , 2015, Nature Communications.

[74]  A. Kawahara,et al.  Efficient Multiple Genome Modifications Induced by the crRNAs, tracrRNA and Cas9 Protein Complex in Zebrafish , 2015, PloS one.

[75]  K. Musunuru,et al.  Novel Genome-Editing Tools to Model and Correct Primary Immunodeficiencies , 2015, Front. Immunol..

[76]  Sarah Geurs,et al.  TALEN-mediated apc mutation in Xenopus tropicalis phenocopies familial adenomatous polyposis , 2015, Oncoscience.

[77]  Hong Wang,et al.  Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9. , 2015, Human molecular genetics.

[78]  Natalia N. Ivanova,et al.  Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells , 2015, Nature Biotechnology.

[79]  G. Pan,et al.  Factor-induced Reprogramming and Zinc Finger Nuclease-aided Gene Targeting Cause Different Genome Instability in β-Thalassemia Induced Pluripotent Stem Cells (iPSCs)* , 2015, The Journal of Biological Chemistry.

[80]  H. Ploegh,et al.  Inhibition of non-homologous end joining increases the efficiency of CRISPR/Cas9-mediated precise [TM: inserted] genome editing , 2015, Nature Biotechnology.

[81]  Yuriko Osakabe,et al.  Genome editing with engineered nucleases in plants. , 2015, Plant & cell physiology.

[82]  G. Mardon,et al.  CRISPR-engineered mosaicism rapidly reveals that loss of Kcnj13 function in mice mimics human disease phenotypes , 2015, Scientific Reports.

[83]  Randall J. Platt,et al.  Therapeutic genome editing: prospects and challenges , 2015, Nature Medicine.

[84]  Masayo Takahashi,et al.  Design of a Tumorigenicity Test for Induced Pluripotent Stem Cell (iPSC)-Derived Cell Products , 2015, Journal of clinical medicine.

[85]  Yong Fan,et al.  Improved hematopoietic differentiation efficiency of gene-corrected beta-thalassemia induced pluripotent stem cells by CRISPR/Cas9 system. , 2014, Stem cells and development.

[86]  X. Gong,et al.  A Novel GJA8 Mutation (p.V44A) Causing Autosomal Dominant Congenital Cataract , 2014, PloS one.

[87]  Huanming Yang,et al.  Generation of outbred Ace2 knockout mice by RNA transfection of TALENs displaying colitis reminiscent pathophysiology and inflammation , 2014, Transgenic Research.

[88]  J. Renaud,et al.  Characterization of Dystrophin Deficient Rats: A New Model for Duchenne Muscular Dystrophy , 2014, PloS one.

[89]  Joana A. Vidigal,et al.  In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system , 2014, Nature.

[90]  Francisco J. Sánchez-Rivera,et al.  Rapid modeling of cooperating genetic events in cancer through somatic genome editing , 2014, Nature.

[91]  R. Gerard,et al.  TALEN-mediated somatic mutagenesis in murine models of cancer. , 2014, Cancer research.

[92]  Robert Langer,et al.  CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling , 2014, Cell.

[93]  Daniel J. Rader,et al.  Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing , 2014, Circulation research.

[94]  Daniel G. Anderson,et al.  Non-viral vectors for gene-based therapy , 2014, Nature Reviews Genetics.

[95]  Hao Yin,et al.  CRISPR-mediated direct mutation of cancer genes in the mouse liver , 2014, Nature.

[96]  Satoshi Ota,et al.  Multiple genome modifications by the CRISPR/Cas9 system in zebrafish , 2014, Genes to cells : devoted to molecular & cellular mechanisms.

[97]  Aviv Regev,et al.  Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing , 2014, Nature Biotechnology.

[98]  E. Lander,et al.  Development and Applications of CRISPR-Cas9 for Genome Engineering , 2014, Cell.

[99]  M. van der Burg,et al.  Targeted Genome Editing in Human Repopulating Hematopoietic Stem Cells , 2014, Nature.

[100]  H. Harn,et al.  Polyglutamine (PolyQ) Diseases: Genetics to Treatments , 2014, Cell transplantation.

[101]  Matthew Meyerson,et al.  Targeted genomic rearrangements using CRISPR/Cas technology , 2014, Nature Communications.

[102]  Wei-Ting Hwang,et al.  Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. , 2014, The New England journal of medicine.

[103]  M. Meisler,et al.  Modeling human epilepsy by TALEN targeting of mouse sodium channel Scn8a , 2014, Genesis.

[104]  S. Younes,et al.  Severe congenital ocular coloboma , 2014, The Pan African medical journal.

[105]  Yun Bai,et al.  A Large Novel Deletion Downstream of PAX6 Gene in a Chinese Family with Ocular Coloboma , 2013, PloS one.

[106]  Rudolf Jaenisch,et al.  One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering , 2013, Cell.

[107]  H. Harn,et al.  Parkinson's Disease: From Genetics to Treatments , 2013, Cell transplantation.

[108]  Wolfgang Wurst,et al.  Direct production of mouse disease models by embryo microinjection of TALENs and oligodeoxynucleotides , 2013, Proceedings of the National Academy of Sciences.

[109]  Seung Woo Cho,et al.  Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease , 2013, Nature Biotechnology.

[110]  Jeffry D. Sander,et al.  Efficient In Vivo Genome Editing Using RNA-Guided Nucleases , 2013, Nature Biotechnology.

[111]  A. Lewin,et al.  Gene therapy in animal models of autosomal dominant retinitis pigmentosa , 2012, Molecular vision.

[112]  D. Voytas,et al.  Efficient TALEN-mediated gene knockout in livestock , 2012, Proceedings of the National Academy of Sciences.

[113]  J. Doudna,et al.  A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity , 2012, Science.

[114]  S. Imbeaud,et al.  Integrated analysis of somatic mutations and focal copy-number changes identifies key genes and pathways in hepatocellular carcinoma , 2012, Nature Genetics.

[115]  Jeffry D. Sander,et al.  FLASH Assembly of TALENs Enables High-Throughput Genome Editing , 2012, Nature Biotechnology.

[116]  F. Bushman,et al.  In vivo genome editing restores hemostasis in a mouse model of hemophilia , 2011, Nature.

[117]  K. High,et al.  Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges , 2011, Nature Reviews Genetics.

[118]  Wei Li,et al.  Generation of PPARγ mono-allelic knockout pigs via zinc-finger nucleases and nuclear transfer cloning , 2011, Cell Research.

[119]  J. X. Jiang,et al.  Gap junctions or hemichannel-dependent and independent roles of connexins in cataractogenesis and lens development. , 2010, Current molecular medicine.

[120]  S. Rodríguez-Perales,et al.  MLL gene fusions in human leukaemias: in vivo modelling to recapitulate these primary tumourigenic events , 2008, International journal of hematology.

[121]  D. Neil Hayes,et al.  LKB1 modulates lung cancer differentiation and metastasis , 2007, Nature.

[122]  G. Lucignani,et al.  Cancer modeling: modern imaging applications in the generation of novel animal model systems to study cancer progression and therapy. , 2007, The international journal of biochemistry & cell biology.

[123]  B. Fehse,et al.  Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors. , 2006, Human gene therapy.

[124]  N. Newman Hereditary optic neuropathies: from the mitochondria to the optic nerve. , 2005, American journal of ophthalmology.

[125]  Adam Bagg,et al.  Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. , 2003, Molecular genetics and metabolism.

[126]  T. Jacks,et al.  Cancer Modeling in the Modern Era Progress and Challenges , 2002, Cell.

[127]  L. Strong,et al.  Positional cloning and characterization of a paired box- and homeobox-containing gene from the aniridia region , 1991, Cell.

[128]  Xiaoping Chen,et al.  Simultaneous Knockout of CXCR4 and CCR5 Genes in CD4+ T Cells via CRISPR/Cas9 Confers Resistance to Both X4- and R5-Tropic Human Immunodeficiency Virus Type 1 Infection. , 2018, Human gene therapy.

[129]  K. Musunuru,et al.  Genome editing in cardiovascular diseases , 2017, Nature Reviews Cardiology.

[130]  E. Lander,et al.  Development and Applications of CRISPR-Cas 9 for Genome Engineering , 2015 .

[131]  N. Zhang,et al.  One-step generation of different immunodeficient mice with multiple gene modifications by CRISPR/Cas9 mediated genome engineering. , 2014, The international journal of biochemistry & cell biology.

[132]  J. Uitto,et al.  Molecular genetics of Meesmann's corneal dystrophy: ancestral and novel mutations in keratin 12 (K12) and complete sequence of the human KRT12 gene. , 2000, Experimental eye research.

[133]  Jeffry D Sander,et al.  FLAsH assembly of TALeNs for high-throughput genome editing , 2022 .