Delivery approaches for CRISPR/Cas9 therapeutics in vivo: advances and challenges

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

(2018) Ying-Li Luo et al.   ACS Nano

Protein delivery into cells using inorganic nanoparticle–protein supramolecular assemblies

(2018) Federica Scaletti et al.   CHEMICAL SOCIETY REVIEWS

Current Status of Nonviral Vectors for Gene Therapy in China

(2018) Li Liu et al.   HUMAN GENE THERAPY

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

(2018) Jonathan D. Finn et al.   Cell Reports

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

Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

(2017) Hao Yin et al.   NATURE BIOTECHNOLOGY

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

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

CRISPR/Cas9 delivery with one single adenoviral vector devoid of all viral genes

(2017) Eric Ehrke-Schulz et al.   Scientific Reports

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

(2016) Ling Li et al.   ACS Nano

Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA

(2016) Jason B. Miller et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Programmable RNA Tracking in Live Cells with CRISPR/Cas9

(2016) David A. Nelles et al.   CELL

Highly Efficient Mouse Genome Editing by CRISPR Ribonucleoprotein Electroporation of Zygotes

(2016) Sean Chen et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Versatility of chemically synthesized guide RNAs for CRISPR-Cas9 genome editing

(2016) Melissa L. Kelley et al.   JOURNAL OF BIOTECHNOLOGY

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

(2016) Daniel P. Dever et al.   NATURE

In vitro and ex vivo strategies for intracellular delivery

(2016) Martin P. Stewart et al.   NATURE

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

In vivo high-throughput profiling of CRISPR–Cpf1 activity

(2016) Hui K Kim et al.   NATURE METHODS

Defining and improving the genome-wide specificities of CRISPR–Cas9 nucleases

(2016) Shengdar Q. Tsai et al.   NATURE REVIEWS GENETICS

Engineering Delivery Vehicles for Genome Editing

(2016) Christopher E. Nelson et al.   Annual Review of Chemical and Biomolecular Engineering

Elimination of HIV-1 Genomes from Human T-lymphoid Cells by CRISPR/Cas9 Gene Editing

(2016) Rafal Kaminski et al.   Scientific Reports

Self-Assembled DNA Nanoclews for the Efficient Delivery of CRISPR-Cas9 for Genome Editing

(2015) Wujin Sun et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

CRISPR genome engineering and viral gene delivery: A case of mutual attraction

(2015) Florian Schmidt et al.   Biotechnology Journal

Pancreatic cancer modeling using retrograde viral vector delivery and in vivo CRISPR/Cas9-mediated somatic genome editing

(2015) Shin-Heng Chiou et al.   GENES & DEVELOPMENT

High Efficiency, Homology-Directed Genome Editing in Caenorhabditis elegans Using CRISPR-Cas9 Ribonucleoprotein Complexes

(2015) A. Paix et al.   GENETICS

Challenges in CRISPR/CAS9 Delivery: Potential Roles of Nonviral Vectors

(2015) Ling Li 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

Expanding the Biologist’s Toolkit with CRISPR-Cas9

(2015) Samuel H. Sternberg et al.   MOLECULAR CELL

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

Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers

(2015) Isaac B Hilton et al.   NATURE BIOTECHNOLOGY

Dynamics of CRISPR-Cas9 genome interrogation in living cells

(2015) Spencer C. Knight et al.   SCIENCE

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

(2015) Marc Zuckermann et al.   Nature Communications

An Inducible Lentiviral Guide RNA Platform Enables the Identification of Tumor-Essential Genes and Tumor-Promoting Mutations In Vivo

(2015) Brandon J. Aubrey et al.   Cell Reports

CRISPR genome engineering and viral gene delivery: A case of mutual attraction

(2015) Florian Schmidt et al.   Biotechnology Journal

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling

(2014) Randall J. Platt et al.   CELL

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

(2014) Yuyu Niu 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

Generation of CRISPR/Cas9-mediated gene-targeted pigs via somatic cell nuclear transfer

(2014) Xiaoqing Zhou et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Efficient gene editing in adult mouse livers via adenoviral delivery of CRISPR/Cas9

(2014) Ranran Cheng et al.   FEBS LETTERS

Highly Specific and Efficient CRISPR/Cas9-Catalyzed Homology-Directed Repair inDrosophila

(2014) Scott J. Gratz et al.   GENETICS

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

Rational design of highly active sgRNAs for CRISPR-Cas9–mediated gene inactivation

(2014) John G Doench et al.   NATURE BIOTECHNOLOGY

Fusion of catalytically inactive Cas9 to FokI nuclease improves the specificity of genome modification

(2014) John P Guilinger et al.   NATURE BIOTECHNOLOGY

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

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

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

(2014) Bin Shen et al.   NATURE METHODS

A guide to genome engineering with programmable nucleases

(2014) Hyongbum Kim et al.   NATURE REVIEWS GENETICS

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Simple and Rapid In Vivo Generation of Chromosomal Rearrangements using CRISPR/Cas9 Technology

(2014) Rafael B. Blasco et al.   Cell Reports

Mechanism of Polyplex- and Lipoplex-Mediated Delivery of Nucleic Acids: Real-Time Visualization of Transient Membrane Destabilization without Endosomal Lysis

(2013) Zia ur Rehman et al.   ACS Nano

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

(2013) Lei S. Qi et al.   CELL

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

(2013) F. Ann Ran et al.   CELL

Cas9 as a versatile tool for engineering biology

(2013) Prashant Mali et al.   NATURE METHODS

CRISPR RNA–guided activation of endogenous human genes

(2013) Morgan L Maeder et al.   NATURE METHODS

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity

(2013) Thomas J. Cradick et al.   NUCLEIC ACIDS RESEARCH

Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells

(2013) Ophir Shalem et al.   SCIENCE

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

(2013) Thomas Gaj et al.   TRENDS IN BIOTECHNOLOGY

Endosomal escape pathways for delivery of biologicals

(2010) Amir K. Varkouhi et al.   JOURNAL OF CONTROLLED RELEASE

HIV-1 Lentiviral Vector Immunogenicity Is Mediated by Toll-Like Receptor 3 (TLR3) and TLR7

(2010) K. Breckpot et al.   JOURNAL OF VIROLOGY

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

(2010) Josiane E. Garneau et al.   NATURE

Progress and prospects: immune responses to viral vectors

(2009) S Nayak et al.   GENE THERAPY

Persistence of High-Capacity Adenoviral Vectors as Replication-Defective Monomeric Genomes In Vitro and in Murine Liver

(2009) Lorenz Jager et al.   HUMAN GENE THERAPY

Gold nanoparticles in delivery applications☆

(2008) P GHOSH et al.   ADVANCED DRUG DELIVERY REVIEWS

Non-Integrating Lentiviral Vectors

(2008) Chamsy Sarkis et al.   CURRENT GENE THERAPY