Programmable Site-Specific Nucleases for Targeted Genome Engineering in Higher Eukaryotes

Efficient targeted mutagenesis in soybean by TALENs and CRISPR/Cas9

(2016) Hongyang Du et al.   JOURNAL OF BIOTECHNOLOGY

Development of broad virus resistance in non-transgenic cucumber using CRISPR/Cas9 technology

(2016) Jeyabharathy Chandrasekaran et al.   MOLECULAR PLANT PATHOLOGY

In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa

(2016) Benjamin Bakondi et al.   MOLECULAR THERAPY

One-step generation of triple gene-targeted pigs using CRISPR/Cas9 system

(2016) Xianlong Wang et al.   Scientific Reports

In situ genetic correction of F8 intron 22 inversion in hemophilia A patient-specific iPSCs

(2016) Yong Wu et al.   Scientific Reports

Precision Medicine: Genetic Repair of Retinitis Pigmentosa in Patient-Derived Stem Cells

(2016) Alexander G. Bassuk et al.   Scientific Reports

CRISPR/Cas9-mediated mutagenesis of the RIN locus that regulates tomato fruit ripening

(2015) Yasuhiro Ito et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

(2015) R. Sharma et al.   BLOOD

Targeted genome modifications in soybean with CRISPR/Cas9

(2015) Thomas B Jacobs et al.   BMC BIOTECHNOLOGY

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

DNA-free genome editing in plants with preassembled CRISPR-Cas9 ribonucleoproteins

(2015) Je Wook Woo et al.   NATURE BIOTECHNOLOGY

Improving cold storage and processing traits in potato through targeted gene knockout

(2015) Benjamin M. Clasen et al.   PLANT BIOTECHNOLOGY JOURNAL

Creation of fragrant rice by targeted knockout of theOsBADH2gene using TALEN technology

(2015) Qiwei Shan et al.   PLANT BIOTECHNOLOGY JOURNAL

Heritable site-specific mutagenesis using TALENs in maize

(2015) Si Nian Char et al.   PLANT BIOTECHNOLOGY JOURNAL

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

Efficient introgression of allelic variants by embryo-mediated editing of the bovine genome

(2015) Jingwei Wei et al.   Scientific Reports

Both TALENs and CRISPR/Cas9 directly target the HBB IVS2–654 (C > T) mutation in β-thalassemia-derived iPSCs

(2015) Peng Xu et al.   Scientific Reports

Genome editing of CXCR4 by CRISPR/cas9 confers cells resistant to HIV-1 infection

(2015) Panpan Hou et al.   Scientific Reports

Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting

(2015) Chia-Wei Chang et al.   Cell Reports

CCR5 Disruption in Induced Pluripotent Stem Cells Using CRISPR/Cas9 Provides Selective Resistance of Immune Cells to CCR5-tropic HIV-1 Virus

(2015) HyunJun Kang et al.   Molecular Therapy-Nucleic Acids

Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9

(2015) Hongmei Lisa Li et al.   Stem Cell Reports

Targeted Correction and Restored Function of the CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells

(2015) Ana M. Crane et al.   Stem Cell Reports

TALEN-Based Mutagenesis of Lipoxygenase LOX3 Enhances the Storage Tolerance of Rice (Oryza sativa) Seeds

(2015) Lei Ma et al.   PLoS One

Generation and Inheritance of Targeted Mutations in Potato (Solanum tuberosum L.) Using the CRISPR/Cas System

(2015) Nathaniel M. Butler et al.   PLoS One

Genome Engineering with Targetable Nucleases

(2014) Dana Carroll   Annual Review of Biochemistry

Use of the CRISPR/Cas9 System to Produce Genetically Engineered Pigs from In Vitro-Derived Oocytes and Embryos1

(2014) Kristin M. Whitworth et al.   BIOLOGY OF REPRODUCTION

One-step generation of knockout pigs by zygote injection of CRISPR/Cas system

(2014) Tang Hai et al.   CELL RESEARCH

Simultaneous editing of three homoeoalleles in hexaploid bread wheat confers heritable resistance to powdery mildew

(2014) Yanpeng Wang et al.   NATURE BIOTECHNOLOGY

Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects

(2014) Bin Shen et al.   NATURE METHODS

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

(2014) Pablo Tebas et al.   NEW ENGLAND JOURNAL OF MEDICINE

Large chromosomal deletions and heritable small genetic changes induced by CRISPR/Cas9 in rice

(2014) Huanbin Zhou et al.   NUCLEIC ACIDS RESEARCH

TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity

(2014) Claudio Mussolino et al.   NUCLEIC ACIDS RESEARCH

Improved soybean oil quality by targeted mutagenesis of the fatty acid desaturase 2 gene family

(2014) William Haun et al.   PLANT BIOTECHNOLOGY JOURNAL

Hairy Root Transformation Using Agrobacterium rhizogenes as a Tool for Exploring Cell Type-Specific Gene Expression and Function Using Tomato as a Model

(2014) M. Ron et al.   PLANT PHYSIOLOGY

Targeted Mutagenesis of the Tomato PROCERA Gene Using Transcription Activator-Like Effector Nucleases

(2014) V. S. Lor et al.   PLANT PHYSIOLOGY

Efficient Gene Editing in Tomato in the First Generation Using the Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-Associated9 System

(2014) C. Brooks et al.   PLANT PHYSIOLOGY

Targeted mutagenesis using zinc-finger nucleases in perennial fruit trees

(2014) Reut Peer et al.   PLANTA

True-Breeding Targeted Gene Knock-Out in Barley Using Designer TALE-Nuclease in Haploid Cells

(2014) Maia Gurushidze et al.   PLoS One

Targeted Genome Editing of Sweet Orange Using Cas9/sgRNA

(2014) Hongge Jia et al.   PLoS One

Seamless correction of the sickle cell disease mutation of theHBBgene in human induced pluripotent stem cells using TALENs

(2013) Ning Sun et al.   BIOTECHNOLOGY AND BIOENGINEERING

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

(2013) Gerald Schwank et al.   Cell Stem Cell

Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9

(2013) Yuxuan Wu et al.   Cell Stem Cell

Enhanced Efficiency of Human Pluripotent Stem Cell Genome Editing through Replacing TALENs with CRISPRs

(2013) Qiurong Ding et al.   Cell Stem Cell

Efficient drug screening and gene correction for treating liver disease using patient-specific stem cells

(2013) Su Mi Choi et al.   HEPATOLOGY

Transcription Activator-like Effector Nuclease (TALEN)-mediated Gene Correction in Integration-free β-Thalassemia Induced Pluripotent Stem Cells

(2013) Ning Ma et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Targeted Mutagenesis in Zea mays Using TALENs and the CRISPR/Cas System

(2013) Zhen Liang et al.   Journal of Genetics and Genomics

Application of the CRISPR–Cas System for Efficient Genome Engineering in Plants

(2013) Yanfei Mao et al.   Molecular Plant

Inactivation of Hepatitis B Virus Replication in Cultured Cells and In Vivo with Engineered Transcription Activator-Like Effector Nucleases

(2013) Kristie Bloom et al.   MOLECULAR THERAPY

Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients

(2013) David G Ousterout et al.   MOLECULAR THERAPY

Targeted genome modification of crop plants using a CRISPR-Cas system

(2013) Qiwei Shan et al.   NATURE BIOTECHNOLOGY

Site- and strand-specific nicking of DNA by fusion proteins derived from MutH and I-SceI or TALE repeats

(2013) Lilia Gabsalilow et al.   NUCLEIC ACIDS RESEARCH

Demonstration of CRISPR/Cas9/sgRNA-mediated targeted gene modification in Arabidopsis, tobacco, sorghum and rice

(2013) Wenzhi Jiang et al.   NUCLEIC ACIDS RESEARCH

Trait stacking via targeted genome editing

(2013) William M. Ainley et al.   PLANT BIOTECHNOLOGY JOURNAL

crRNA and tracrRNA guide Cas9-mediated DNA interference inStreptococcus thermophilus

(2013) Tautvydas Karvelis et al.   RNA Biology

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

RNA-dependent DNA endonuclease Cas9 of the CRISPR system: Holy Grail of genome editing?

(2013) Giedrius Gasiunas et al.   TRENDS IN MICROBIOLOGY

Donor DNA Utilization During Gene Targeting with Zinc-Finger Nucleases

(2013) Kelly J. Beumer et al.   G3-Genes Genomes Genetics

RNA-Guided Genome Editing for Target Gene Mutations in Wheat

(2013) Santosh Kumar Upadhyay et al.   G3-Genes Genomes Genetics

Zinc-finger nuclease-mediated correction of  -thalassemia in iPS cells

(2012) C.-J. Chang et al.   BLOOD

An Inhibitor of Nonhomologous End-Joining Abrogates Double-Strand Break Repair and Impedes Cancer Progression

(2012) Mrinal Srivastava et al.   CELL

Precision genome engineering with programmable DNA-nicking enzymes

(2012) E. Kim et al.   GENOME RESEARCH

Playing the End Game: DNA Double-Strand Break Repair Pathway Choice

(2012) J. Ross Chapman et al.   MOLECULAR CELL

High-efficiency TALEN-based gene editing produces disease-resistant rice

(2012) Ting Li et al.   NATURE BIOTECHNOLOGY

Engineered zinc finger nickases induce homology-directed repair with reduced mutagenic effects

(2012) Cherie L. Ramirez et al.   NUCLEIC ACIDS RESEARCH

Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria

(2012) G. Gasiunas et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Generation and Genetic Engineering of Human Induced Pluripotent Stem Cells Using Designed Zinc Finger Nucleases

(2012) Sivaprakash Ramalingam et al.   STEM CELLS AND DEVELOPMENT

Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease

(2011) J. Zou et al.   BLOOD

Generation of Isogenic Pluripotent Stem Cells Differing Exclusively at Two Early Onset Parkinson Point Mutations

(2011) Frank Soldner et al.   CELL

Highly efficient modification of beta-lactoglobulin (BLG) gene via zinc-finger nucleases in cattle

(2011) Shengli Yu et al.   CELL RESEARCH

Targeted chromosomal duplications and inversions in the human genome using zinc finger nucleases

(2011) H. J. Lee et al.   GENOME RESEARCH

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

Genetic engineering of human pluripotent cells using TALE nucleases

(2011) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

An unbiased genome-wide analysis of zinc-finger nuclease specificity

(2011) Richard Gabriel et al.   NATURE BIOTECHNOLOGY

High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases

(2011) Fuqiang Chen et al.   NATURE METHODS

Targeted Mutagenesis of Duplicated Genes in Soybean with Zinc-Finger Nucleases

(2011) S. J. Curtin et al.   PLANT PHYSIOLOGY

In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases

(2011) Vittorio Sebastiano et al.   STEM CELLS

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

(2010) M. Christian et al.   GENETICS

Creating Designed Zinc-Finger Nucleases with Minimal Cytotoxicity

(2010) Sivaprakash Ramalingam et al.   JOURNAL OF MOLECULAR BIOLOGY

Frequent Endonuclease Cleavage at Off-target Locations In Vivo

(2010) Lisa M Petek et al.   MOLECULAR THERAPY

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

(2010) Nathalia Holt et al.   NATURE BIOTECHNOLOGY

Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology

(2010) Salvatore J. Orlando et al.   NUCLEIC ACIDS RESEARCH

Precise genome modification in the crop species Zea mays using zinc-finger nucleases

(2009) Vipula K. Shukla et al.   NATURE

The promises and pitfalls of RNA-interference-based therapeutics

(2009) Daniela Castanotto et al.   NATURE

Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application

(2009) Aimee L. Jackson et al.   NATURE REVIEWS DRUG DISCOVERY

Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors

(2009) J. Boch et al.   SCIENCE

A Simple Cipher Governs DNA Recognition by TAL Effectors

(2009) M. J. Moscou et al.   SCIENCE

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

(2008) Elena E Perez et al.   NATURE BIOTECHNOLOGY