Delivery of CRISPR/Cas9 for therapeutic genome editing

Material solutions for delivery of CRISPR/Cas-based genome editing tools: Current status and future outlook

(2019) Tao Wan et al.   Materials Today

A boronic acid–rich dendrimer with robust and unprecedented efficiency for cytosolic protein delivery and CRISPR-Cas9 gene editing

(2019) Chongyi Liu et al.   Science Advances

Macrophage-Specific in Vivo Gene Editing Using Cationic Lipid-Assisted Polymeric Nanoparticles

(2018) Ying-Li Luo et al.   ACS Nano

Thermo-triggered Release of CRISPR-Cas9 System by Lipid-Encapsulated Gold Nanoparticles for Tumor Therapy

(2018) Peng Wang et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Optimization of lipid-assisted nanoparticle for disturbing neutrophils-related inflammation

(2018) Yang Liu et al.   BIOMATERIALS

Non-viral delivery systems for CRISPR/Cas9-based genome editing: Challenges and opportunities

(2018) Ling Li et al.   BIOMATERIALS

Transcriptome Engineering with RNA-Targeting Type VI-D CRISPR Effectors

(2018) Silvana Konermann et al.   CELL

Cationic Polymer-Mediated CRISPR/Cas9 Plasmid Delivery for Genome Editing

(2018) Zhen Zhang et al.   MACROMOLECULAR RAPID COMMUNICATIONS

A CRISPR/molecular beacon hybrid system for live-cell genomic imaging

(2018) Xiaotian Wu et al.   NUCLEIC ACIDS RESEARCH

Nonviral gene editing via CRISPR/Cas9 delivery by membrane-disruptive and endosomolytic helical polypeptide

(2018) Hong-Xia Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Engineering the Delivery System for CRISPR-Based Genome Editing

(2018) Zachary Glass et al.   TRENDS IN BIOTECHNOLOGY

HPV Oncogene Manipulation Using Nonvirally Delivered CRISPR/Cas9 or Natronobacterium gregoryi Argonaute

(2018) Yeh-Hsing Lao et al.   Advanced Science

Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours

(2018) Bumwhee Lee et al.   Nature Biomedical Engineering

Multifunctional Vector for Delivery of Genome Editing Plasmid Targeting β-Catenin to Remodulate Cancer Cell Properties

(2018) Xiao-Yan He et al.   ACS Applied Materials & Interfaces

Targeting of NLRP3 inflammasome with gene editing for the amelioration of inflammatory diseases

(2018) Congfei Xu et al.   Nature Communications

Bioreducible branched poly(modified nona-arginine) cell-penetrating peptide as a novel gene delivery platform

(2017) Jisang Yoo et al.   JOURNAL OF CONTROLLED RELEASE

Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications

(2017) Chang Liu et al.   JOURNAL OF CONTROLLED RELEASE

Cas13b Is a Type VI-B CRISPR-Associated RNA-Guided RNase Differentially Regulated by Accessory Proteins Csx27 and Csx28

(2017) Aaron A. Smargon et al.   MOLECULAR CELL

Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

(2017) Xue Gao et al.   NATURE

Diversity and evolution of class 2 CRISPR–Cas systems

(2017) Sergey Shmakov et al.   NATURE REVIEWS MICROBIOLOGY

Leveraging Physiology for Precision Drug Delivery

(2017) Wujin Sun et al.   PHYSIOLOGICAL REVIEWS

Cell-Penetrating Peptides: From Basic Research to Clinics

(2017) Giulia Guidotti et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Peptide/Cas9 nanostructures for ribonucleoprotein cell membrane transport and gene edition

(2017) Irene Lostalé-Seijo et al.   Chemical Science

Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice

(2017) Wenhan Yu et al.   Nature Communications

Genome Editing for Cancer Therapy: Delivery of Cas9 Protein/sgRNA Plasmid via a Gold Nanocluster/Lipid Core-Shell Nanocarrier

(2017) Peng Wang et al.   Advanced Science

Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

Artificial Virus Delivers CRISPR-Cas9 System for Genome Editing of Cells in Mice

(2016) Ling Li et al.   ACS Nano

Engineered Viruses as Genome Editing Devices

(2016) Xiaoyu Chen et al.   MOLECULAR THERAPY

CRISPR gene-editing tested in a person for the first time

(2016) David Cyranoski   NATURE

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

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Diverse evolutionary roots and mechanistic variations of the CRISPR-Cas systems

(2016) Prarthana Mohanraju et al.   SCIENCE

Physical Principles of Nanoparticle Cellular Endocytosis

(2015) Sulin Zhang et al.   ACS Nano

Viral Vectors for Gene Therapy: Translational and Clinical Outlook

(2015) Melissa A. Kotterman et al.   Annual Review of Biomedical Engineering

Genome-wide CRISPR Screen in a Mouse Model of Tumor Growth and Metastasis

(2015) Sidi Chen et al.   CELL

Crystal Structure of Staphylococcus aureus Cas9

(2015) Hiroshi Nishimasu et al.   CELL

Efficient CRISPR/Cas9-Mediated Genome Editing in Mice by Zygote Electroporation of Nuclease

(2015) Wenning Qin et al.   GENETICS

Delivery and Specificity of CRISPR/Cas9 Genome Editing Technologies for Human Gene Therapy

(2015) Jennifer L. Gori et al.   HUMAN GENE THERAPY

Adenovirus-Mediated Somatic Genome Editing ofPtenby CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses

(2015) Dan Wang et al.   HUMAN GENE THERAPY

Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection

(2015) Xiquan Liang et al.   JOURNAL OF BIOTECHNOLOGY

CRISPR-Cas9: a new and promising player in gene therapy

(2015) Lu Xiao-Jie et al.   JOURNAL OF MEDICAL GENETICS

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

(2015) Taeyoung Koo et al.   MOLECULES AND CELLS

Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex

(2015) Silvana Konermann et al.   NATURE

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

Delivering the goods: Scientists seek a way to make CRISPR-Cas gene editing more targeted

(2015) Amanda B Keener   NATURE MEDICINE

Modeling colorectal cancer using CRISPR-Cas9–mediated engineering of human intestinal organoids

(2015) Mami Matano et al.   NATURE MEDICINE

High-throughput functional genomics using CRISPR–Cas9

(2015) Ophir Shalem et al.   NATURE REVIEWS GENETICS

An updated evolutionary classification of CRISPR–Cas systems

(2015) Kira S. Makarova et al.   NATURE REVIEWS MICROBIOLOGY

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

(2015) C. Long et al.   SCIENCE

Somatic CRISPR/Cas9-mediated tumour suppressor disruption enables versatile brain tumour modelling

(2015) Marc Zuckermann et al.   Nature Communications

Efficient CRISPR/Cas9-Mediated Genome Editing in Mice by Zygote Electroporation of Nuclease

(2015) Wenning Qin et al.   GENETICS

CRISPR-Cas9 delivery to hard-to-transfect cells via membrane deformation

(2015) X. Han et al.   Science Advances

Platelet-like Nanoparticles: Mimicking Shape, Flexibility, and Surface Biology of Platelets To Target Vascular Injuries

(2014) Aaron C. Anselmo et al.   ACS Nano

CRISPR/Cas9-mediated genome engineering: An adeno-associated viral (AAV) vector toolbox

(2014) Elena Senís et al.   Biotechnology Journal

Generation of Gene-Modified Cynomolgus Monkey via Cas9/RNA-Mediated Gene Targeting in One-Cell Embryos

(2014) Yuyu Niu et al.   CELL

Correction of a genetic disease by CRISPR-Cas9-mediated gene editing in mouse spermatogonial stem cells

(2014) Yuxuan Wu et al.   CELL RESEARCH

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

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

Gene disruption by cell-penetrating peptide-mediated delivery of Cas9 protein and guide RNA

(2014) S. Ramakrishna 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-Cas Systems: Prokaryotes Upgrade to Adaptive Immunity

(2014) Rodolphe Barrangou et al.   MOLECULAR CELL

High-throughput screening of a CRISPR/Cas9 library for functional genomics in human cells

(2014) Yuexin Zhou et al.   NATURE

In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system

(2014) Danilo Maddalo et al.   NATURE

CRISPR-mediated direct mutation of cancer genes in the mouse liver

(2014) Wen Xue et al.   NATURE

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

CRISPR-Cas systems for editing, regulating and targeting genomes

(2014) Jeffry D Sander et al.   NATURE BIOTECHNOLOGY

Exploiting CRISPR-Cas nucleases to produce sequence-specific antimicrobials

(2014) David Bikard et al.   NATURE BIOTECHNOLOGY

Extracellular matrix stiffness and composition jointly regulate the induction of malignant phenotypes in mammary epithelium

(2014) Ovijit Chaudhuri et al.   NATURE MATERIALS

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

(2014) Bin Shen et al.   NATURE METHODS

Unravelling the structural and mechanistic basis of CRISPR–Cas systems

(2014) John van der Oost et al.   NATURE REVIEWS MICROBIOLOGY

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

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

TRAIL-coated leukocytes that kill cancer cells in the circulation

(2014) M. J. Mitchell et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

To acquire or resist: the complex biological effects of CRISPR–Cas systems

(2014) Joseph Bondy-Denomy et al.   TRENDS IN MICROBIOLOGY

Targeted genomic rearrangements using CRISPR/Cas technology

(2014) Peter S. Choi et al.   Nature Communications

A CRISPR/Cas9-Based System for Reprogramming Cell Lineage Specification

(2014) Syandan Chakraborty et al.   Stem Cell Reports

One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering

(2013) Haoyi Wang et al.   CELL

Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression

(2013) Lei S. Qi et al.   CELL

Dynamic Imaging of Genomic Loci in Living Human Cells by an Optimized CRISPR/Cas System

(2013) Baohui Chen et al.   CELL

CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes

(2013) Luke A. Gilbert et al.   CELL

Genome editing with RNA-guided Cas9 nuclease in Zebrafish embryos

(2013) Nannan Chang et al.   CELL RESEARCH

Controlled pH Stability and Adjustable Cellular Uptake of Mixed-Charge Nanoparticles

(2013) Pramod P. Pillai et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Optical control of mammalian endogenous transcription and epigenetic states

(2013) Silvana Konermann et al.   NATURE

Genome-wide recessive genetic screening in mammalian cells with a lentiviral CRISPR-guide RNA library

(2013) Hiroko Koike-Yusa et al.   NATURE BIOTECHNOLOGY

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

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

Targeted mutagenesis in the model plant Nicotiana benthamiana using Cas9 RNA-guided endonuclease

(2013) Vladimir Nekrasov et al.   NATURE BIOTECHNOLOGY

Efficient genome editing in zebrafish using a CRISPR-Cas system

(2013) Woong Y Hwang et al.   NATURE BIOTECHNOLOGY

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Rapid formation of plasma protein corona critically affects nanoparticle pathophysiology

(2013) Stefan Tenzer et al.   Nature Nanotechnology

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Genome engineering in Saccharomyces cerevisiae using CRISPR-Cas systems

(2013) James E. DiCarlo et al.   NUCLEIC ACIDS RESEARCH

Efficient genome engineering in human pluripotent stem cells using Cas9 from Neisseria meningitidis

(2013) Z. Hou et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A vector-free microfluidic platform for intracellular delivery

(2013) A. Sharei et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Minimal "Self" Peptides That Inhibit Phagocytic Clearance and Enhance Delivery of Nanoparticles

(2013) P. L. Rodriguez et al.   SCIENCE

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells

(2013) Ophir Shalem et al.   SCIENCE

Genetic Screens in Human Cells Using the CRISPR-Cas9 System

(2013) Tim Wang et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Targeting receptor-mediated endocytotic pathways with nanoparticles: Rationale and advances

(2012) Shi Xu et al.   ADVANCED DRUG DELIVERY REVIEWS

DNA aptamer functionalized nanomaterials for intracellular analysis, cancer cell imaging and drug delivery

(2012) Hang Xing et al.   CURRENT OPINION IN CHEMICAL BIOLOGY

Novel Protein Transduction Domain Mimics as Nonviral Delivery Vectors for siRNA Targeting NOTCH1 in Primary Human T cells

(2012) A. Özgül Tezgel et al.   MOLECULAR THERAPY

The Possible “Proton Sponge ” Effect of Polyethylenimine (PEI) Does Not Include Change in Lysosomal pH

(2012) Rikke V Benjaminsen et al.   MOLECULAR THERAPY

RNA-guided genetic silencing systems in bacteria and archaea

(2012) Blake Wiedenheft et al.   NATURE

A simple and rapid nonviral approach to efficiently transfect primary tissue–derived cells using polyethylenimine

(2012) Charlie Yu Ming Hsu et al.   Nature Protocols

Homologous recombination and its regulation

(2012) L. Krejci 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

Memory of viral infections by CRISPR-Cas adaptive immune systems: Acquisition of new information

(2012) Peter C. Fineran et al.   VIROLOGY

Antibacterial Studies of Cationic Polymers with Alternating, Random, and Uniform Backbones

(2011) Airong Song et al.   ACS Chemical Biology

Unification of Cas protein families and a simple scenario for the origin and evolution of CRISPR-Cas systems

(2011) Kira S Makarova et al.   Biology Direct

De Novo Designed Protein Transduction Domain Mimics from Simple Synthetic Polymers

(2011) A. Özgül Tezgel et al.   BIOMACROMOLECULES

Tailoring nanocarriers for intracellular protein delivery

(2011) Zhen Gu et al.   CHEMICAL SOCIETY REVIEWS

Influence of Cationic Lipid Composition on Gene Silencing Properties of Lipid Nanoparticle Formulations of siRNA in Antigen-Presenting Cells

(2011) Genc Basha et al.   MOLECULAR THERAPY

CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III

(2011) Elitza Deltcheva et al.   NATURE

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

(2011) Richard Gabriel et al.   NATURE BIOTECHNOLOGY

The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli

(2011) Rimantas Sapranauskas et al.   NUCLEIC ACIDS RESEARCH

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

(2010) M. Christian et al.   GENETICS

Endocytosis of nanomedicines

(2010) Gaurav Sahay et al.   JOURNAL OF CONTROLLED RELEASE

Recent Advances in Lentiviral Vector Development and Applications

(2010) Janka Mátrai et al.   MOLECULAR THERAPY

The CRISPR/Cas bacterial immune system cleaves bacteriophage and plasmid DNA

(2010) Josiane E. Garneau et al.   NATURE

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Exome Sequencing,ANGPTL3Mutations, and Familial Combined Hypolipidemia

(2010) Kiran Musunuru et al.   NEW ENGLAND JOURNAL OF MEDICINE

Coadministration of a Tumor-Penetrating Peptide Enhances the Efficacy of Cancer Drugs

(2010) K. N. Sugahara et al.   SCIENCE

CRISPR/Cas, the Immune System of Bacteria and Archaea

(2010) P. Horvath et al.   SCIENCE

Ultralow-Fouling, Functionalizable, and Hydrolyzable Zwitterionic Materials and Their Derivatives for Biological Applications

(2009) Shaoyi Jiang et al.   ADVANCED MATERIALS

Mechanism of Eukaryotic Homologous Recombination

(2008) Joseph San Filippo et al.   Annual Review of Biochemistry