Therapeutic applications of CRISPR RNA-guided genome editing

CRISPR-Cas9 Targeting ofPCSK9in Human Hepatocytes In Vivo—Brief ReportSignificance

(2016) Xiao Wang et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Effects of genetic correction on the differentiation of hair cell-like cells from iPSCs with MYO15A mutation

(2016) J-R Chen et al.   CELL DEATH AND DIFFERENTIATION

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

(2016) Courtney S. Young et al.   Cell Stem Cell

Genome-wide target specificities of CRISPR-Cas9 nucleases revealed by multiplex Digenome-seq

(2016) Daesik Kim et al.   GENOME RESEARCH

CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice

(2016) Li Xu et al.   MOLECULAR THERAPY

Precise Correction of Disease Mutations in Induced Pluripotent Stem Cells Derived From Patients With Limb Girdle Muscular Dystrophy

(2016) Soeren Turan et al.   MOLECULAR THERAPY

CRISPR-Cas9 Can Inhibit HIV-1 Replication but NHEJ Repair Facilitates Virus Escape

(2016) Gang Wang et al.   MOLECULAR THERAPY

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

(2016) Yang Yang et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

Inhibition of HSV-1 Replication by Gene Editing Strategy

(2016) Pamela C. Roehm et al.   Scientific Reports

Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9

(2015) Chul-Yong Park et al.   Cell Stem Cell

Efficient and Allele-Specific Genome Editing of Disease Loci in Human iPSCs

(2015) Cory Smith et al.   MOLECULAR THERAPY

Measuring and Reducing Off-Target Activities of Programmable Nucleases Including CRISPR-Cas9

(2015) Taeyoung Koo et al.   MOLECULES AND CELLS

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells

(2015) Van Trung Chu et al.   NATURE BIOTECHNOLOGY

A split-Cas9 architecture for inducible genome editing and transcription modulation

(2015) Bernd Zetsche et al.   NATURE BIOTECHNOLOGY

Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

(2015) Daesik Kim et al.   NATURE METHODS

Generation of knock-in primary human T cells using Cas9 ribonucleoproteins

(2015) Kathrin Schumann et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Rational design of a split-Cas9 enzyme complex

(2015) Addison V. Wright et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Modeling Disease In Vivo With CRISPR/Cas9

(2015) Lukas E. Dow   TRENDS IN MOLECULAR MEDICINE

CRISPR/Cas9 cleavage of viral DNA efficiently suppresses hepatitis B virus

(2015) Vyas Ramanan et al.   Scientific Reports

CRISPR/Cas9 nickase-mediated disruption of hepatitis B virus open reading frame S and X

(2015) Madina Karimova 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

Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs

(2015) Amy L. Firth et al.   Cell Reports

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

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

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

CRISPR/Cas9 System as an Agent for Eliminating Polyomavirus JC Infection

(2015) Hassen S. Wollebo et al.   PLoS One

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling

(2014) Randall J. Platt et al.   CELL

Efficient Ablation of Genes in Human Hematopoietic Stem and Effector Cells using CRISPR/Cas9

(2014) Pankaj K. Mandal et al.   Cell Stem Cell

Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 andpiggyBac

(2014) Fei Xie et al.   GENOME RESEARCH

Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins

(2014) S. Kim et al.   GENOME RESEARCH

CRISPR/Cas9-mediated genome editing of Epstein-Barr virus in human cells

(2014) K.-S. Yuen et al.   JOURNAL OF GENERAL VIROLOGY

Inactivation of the Human Papillomavirus E6 or E7 Gene in Cervical Carcinoma Cells by Using a Bacterial CRISPR/Cas RNA-Guided Endonuclease

(2014) E. M. Kennedy et al.   JOURNAL OF VIROLOGY

Sequence-specific antimicrobials using efficiently delivered RNA-guided nucleases

(2014) Robert J Citorik et al.   NATURE BIOTECHNOLOGY

In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

(2014) John A Zuris et al.   NATURE BIOTECHNOLOGY

Exploiting CRISPR-Cas nucleases to produce sequence-specific antimicrobials

(2014) David Bikard et al.   NATURE BIOTECHNOLOGY

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

A guide to genome engineering with programmable nucleases

(2014) Hyongbum Kim et al.   NATURE REVIEWS GENETICS

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

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

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

(2014) C. Long et al.   SCIENCE

Programmable Removal of Bacterial Strains by Use of Genome-Targeting CRISPR-Cas Systems

(2014) A. A. Gomaa et al.   mBio

The CRISPR/Cas9 System Facilitates Clearance of the Intrahepatic HBV Templates In Vivo

(2014) Su-Ru Lin et al.   Molecular Therapy-Nucleic Acids

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

(2013) Yuxuan Wu et al.   Cell Stem Cell

Clinical Trials Using Antisense Oligonucleotides in Duchenne Muscular Dystrophy

(2013) Taeyoung Koo et al.   HUMAN GENE THERAPY

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

RNA-guided editing of bacterial genomes using CRISPR-Cas systems

(2013) Wenyan Jiang et al.   NATURE BIOTECHNOLOGY

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

(2013) Rebecca J. Fairclough et al.   NATURE REVIEWS GENETICS

Mice Transgenic for CD4-Specific Human CD4, CCR5 and Cyclin T1 Expression: A New Model for Investigating HIV-1 Transmission and Treatment Efficacy

(2013) Kieran Seay et al.   PLoS One

Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus

(2013) Hirotaka Ebina et al.   Scientific Reports

CRISPR Interference Can Prevent Natural Transformation and Virulence Acquisition during In Vivo Bacterial Infection

(2012) David Bikard et al.   Cell Host & Microbe

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

(2012) M. Jinek et al.   SCIENCE

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Targeted chromosomal deletions in human cells using zinc finger nucleases

(2009) H. J. Lee et al.   GENOME RESEARCH

Targeted genome editing in human cells with zinc finger nucleases constructed via modular assembly

(2009) H. J. Kim et al.   GENOME RESEARCH

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

Interleukin‐4–Transgenic hu‐PBL‐SCID Mice: A Model for the Screening of Antiviral Drugs and Immunotherapeutic Agents against X4 HIV‐1 Viruses

(2007) Kazu Okuma et al.   JOURNAL OF INFECTIOUS DISEASES