CRISPR/Cas9: Principle, Applications, and Delivery through Extracellular Vesicles

CRISPR technologies for precise epigenome editing

(2021) Muneaki Nakamura et al.   NATURE CELL BIOLOGY

Natural or Synthetic RNA Delivery: A Stoichiometric Comparison of Extracellular Vesicles and Synthetic Nanoparticles

(2021) Daniel E. Murphy et al.   NANO LETTERS

Recent Advancement and Technical Challenges in Developing Small Extracellular Vesicles for Cancer Drug Delivery

(2021) Tianjiao Geng et al.   PHARMACEUTICAL RESEARCH

Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3

(2021) Min Qiu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Next-Generation Cancer Biomarkers: Extracellular Vesicle DNA as a Circulating Surrogate of Tumor DNA

(2021) Samuel Amintas et al.   Frontiers in Cell and Developmental Biology

Extracellular Vesicle-Associated miRNAs as a Biomarker for Lung Cancer in Liquid Biopsy

(2021) Xue He et al.   Frontiers in Molecular Biosciences

Genome-wide programmable transcriptional memory by CRISPR-based epigenome editing

(2021) James K. Nuñez et al.   CELL

Secreted Extracellular Vesicle Molecular Cargo as a Novel Liquid Biopsy Diagnostics of Central Nervous System Diseases

(2021) Sara Monteiro-Reis et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

CRISPR-Cas9 cytidine and adenosine base editing of splice-sites mediates highly-efficient disruption of proteins in primary and immortalized cells

(2021) Mitchell G. Kluesner et al.   Nature Communications

Engineered extracellular vesicles as versatile ribonucleoprotein delivery vehicles for efficient and safe CRISPR genome editing

(2021) Xingang Yao et al.   Journal of Extracellular Vesicles

Programmable base editing of mutated TERT promoter inhibits brain tumour growth

(2020) Xinjian Li et al.   NATURE CELL BIOLOGY

Dual base editor catalyzes both cytosine and adenine base conversions in human cells

(2020) Xiaohui Zhang et al.   NATURE BIOTECHNOLOGY

An MXD1-derived repressor peptide identifies noncoding mediators of MYC-driven cell proliferation

(2020) Philipp Raffeiner et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene-edited human stem cell–derived β cells from a patient with monogenic diabetes reverse preexisting diabetes in mice

(2020) Kristina G. Maxwell et al.   Science Translational Medicine

Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping

(2020) Peter Gee et al.   Nature Communications

Epithelial cell -derived microvesicles: A safe delivery platform of CRISPR/Cas9 conferring synergistic anti-tumor effect with sorafenib

(2020) Cong He et al.   EXPERIMENTAL CELL RESEARCH

Single C-to-T substitution using engineered APOBEC3G-nCas9 base editors with minimum genome- and transcriptome-wide off-target effects

(2020) Sangsin Lee et al.   Science Advances

ACBE, a new base editor for simultaneous C-to-T and A-to-G substitutions in mammalian systems

(2020) Jingke Xie et al.   BMC BIOLOGY

Tropism-facilitated delivery of CRISPR/Cas9 system with chimeric antigen receptor-extracellular vesicles against B-cell malignancies

(2020) Qian Xu et al.   JOURNAL OF CONTROLLED RELEASE

Extracellular vesicles engineered with valency-controlled DNA nanostructures deliver CRISPR/Cas9 system for gene therapy

(2020) Jialang Zhuang et al.   NUCLEIC ACIDS RESEARCH

In vivo CRISPR/Cas9 targeting of fusion oncogenes for selective elimination of cancer cells

(2020) M. Martinez-Lage et al.   Nature Communications

Ready for Repair? Gene Editing Enters the Clinic for the Treatment of Human Disease

(2020) Martijn P.T. Ernst et al.   Molecular Therapy-Methods & Clinical Development

Recent Advances in Nanocarrier-Assisted Therapeutics Delivery Systems

(2020) Shi Su et al.   Pharmaceutics

Hepatic stellate cell reprogramming via exosome-mediated CRISPR/dCas9-VP64 delivery

(2020) Nianan Luo et al.   DRUG DELIVERY

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

Identification of preexisting adaptive immunity to Cas9 proteins in humans

(2019) Carsten T. Charlesworth et al.   NATURE MEDICINE

Delivery Aspects of CRISPR/Cas for in Vivo Genome Editing

(2019) Danny Wilbie et al.   ACCOUNTS OF CHEMICAL RESEARCH

The next generation of CRISPR–Cas technologies and applications

(2019) Adrian Pickar-Oliver et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Ezh2-dCas9 and KRAB-dCas9 enable engineering of epigenetic memory in a context-dependent manner

(2019) Henriette O’Geen et al.   Epigenetics & Chromatin

Gene Editing: Friend or Foe? Evidence Indicates Endogenous Exosomes Can Deliver Functional gRNA and Cas9 Protein (Small 38/2019)

(2019) Ran Chen et al.   Small

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

CRISPRCasdb a successor of CRISPRdb containing CRISPR arrays and cas genes from complete genome sequences, and tools to download and query lists of repeats and spacers

(2019) Christine Pourcel et al.   NUCLEIC ACIDS RESEARCH

High levels of AAV vector integration into CRISPR-induced DNA breaks

(2019) Killian S. Hanlon et al.   Nature Communications

Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII

(2019) Hainan Chen et al.   Scientific Reports

CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis

(2019) Dong Hyun Jo et al.   Science Advances

Methods for the Determination of the Purity of Exosomes

(2019) Katarzyna Kluszczyńska et al.   CURRENT PHARMACEUTICAL DESIGN

CRISPR RNAs trigger innate immune responses in human cells

(2018) Sojung Kim et al.   GENOME RESEARCH

An enhanced CRISPR repressor for targeted mammalian gene regulation

(2018) Nan Cher Yeo et al.   NATURE METHODS

Efficient RNA drug delivery using red blood cell extracellular vesicles

(2018) Waqas Muhammad Usman et al.   Nature Communications

Exosome-Liposome Hybrid Nanoparticles Deliver CRISPR/Cas9 System in MSCs

(2018) Yao Lin et al.   Advanced Science

FDA approves landmark RNAi drug

(2018) Asher Mullard   NATURE REVIEWS DRUG DISCOVERY

Gesicle-Mediated Delivery of CRISPR/Cas9 Ribonucleoprotein Complex for Inactivating the HIV Provirus

(2018) Lee A. Campbell et al.   MOLECULAR THERAPY

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

(2018) Annalisa Lattanzi et al.   MOLECULAR THERAPY

In Vitro and in Vivo RNA Inhibition by CD9-HuR Functionalized Exosomes Encapsulated with miRNA or CRISPR/dCas9

(2018) Zhelong Li et al.   NANO LETTERS

High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population

(2018) Dimitrios L. Wagner et al.   NATURE MEDICINE

Delivery of an Artificial Transcription Regulator dCas9-VPR by Extracellular Vesicles for Therapeutic Gene Activation

(2018) Duško Lainšček et al.   ACS Synthetic Biology

The Methods of Choice for Extracellular Vesicles (EVs) Characterization

(2017) Rafal Szatanek et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Cancer-derived exosomes as a delivery platform of CRISPR/Cas9 confer cancer cell tropism-dependent targeting

(2017) Seung Min Kim et al.   JOURNAL OF CONTROLLED RELEASE

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

MYC Deregulation in Primary Human Cancers

(2017) Manpreet Kalkat et al.   Genes

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

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

CRISPRs for Optimal Targeting: Delivery of CRISPR Components as DNA, RNA, and Protein into Cultured Cells and Single-Cell Embryos

(2016) Evguenia Kouranova et al.   HUMAN GENE THERAPY

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

(2016) Daniel P. Dever et al.   NATURE

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems

(2016) K. Nishida et al.   SCIENCE

Mechanisms of RNA loading into exosomes

(2015) Teresa Janas et al.   FEBS LETTERS

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

Exosomes as drug delivery vehicles for Parkinson's disease therapy

(2015) Matthew J. Haney et al.   JOURNAL OF CONTROLLED RELEASE

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

(2015) Mami Matano et al.   NATURE MEDICINE

Regulated delivery of molecular cargo to invasive tumour-derived microvesicles

(2015) James W. Clancy et al.   Nature Communications

Extracellular microvesicles and invadopodia mediate non-overlapping modes of tumor cell invasion

(2015) Alanna E. Sedgwick et al.   Scientific Reports

Biogenesis, Secretion, and Intercellular Interactions of Exosomes and Other Extracellular Vesicles

(2014) Marina Colombo et al.   Annual Review of Cell and Developmental Biology

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

(2014) S. Kim et al.   GENOME RESEARCH

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

Hypoxia-inducible factors and RAB22A mediate formation of microvesicles that stimulate breast cancer invasion and metastasis

(2014) T. Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases

(2014) Yujia Cai et al.   eLife

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

(2013) Lei S. Qi et al.   CELL

RNA-guided gene activation by CRISPR-Cas9–based transcription factors

(2013) Pablo Perez-Pinera et al.   NATURE METHODS

CRISPR RNA–guided activation of endogenous human genes

(2013) Morgan L Maeder et al.   NATURE METHODS

Extracellular vesicles: biology and emerging therapeutic opportunities

(2013) Samir EL Andaloussi et al.   NATURE REVIEWS DRUG DISCOVERY

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

Sumoylated hnRNPA2B1 controls the sorting of miRNAs into exosomes through binding to specific motifs

(2013) Carolina Villarroya-Beltri et al.   Nature Communications

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

(2012) M. Jinek et al.   SCIENCE

Interaction and uptake of exosomes by ovarian cancer cells

(2011) Cristina Escrevente et al.   BMC CANCER

Protein Transfer Into Human Cells by VSV-G-induced Nanovesicles

(2011) Philippe-Emmanuel Mangeot et al.   MOLECULAR THERAPY

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

(2011) Elitza Deltcheva et al.   NATURE

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

ARF6-Regulated Shedding of Tumor Cell-Derived Plasma Membrane Microvesicles

(2009) Vandhana Muralidharan-Chari et al.   CURRENT BIOLOGY

Glioblastoma microvesicles transport RNA and proteins that promote tumour growth and provide diagnostic biomarkers

(2008) Johan Skog et al.   NATURE CELL BIOLOGY

Small CRISPR RNAs Guide Antiviral Defense in Prokaryotes

(2008) S. J. J. Brouns et al.   SCIENCE