All‐In‐One Dendrimer‐Based Lipid Nanoparticles Enable Precise HDR‐Mediated Gene Editing In Vivo

Hydrophobic Domain Structure of Linear-Dendritic Poly(ethylene glycol) Lipids Affects RNA Delivery of Lipid Nanoparticles

(2020) Kejin Zhou et al.   MOLECULAR PHARMACEUTICS

Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

(2020) Qiang Cheng et al.   Nature Nanotechnology

Delivery of Tissue-Targeted Scalpels: Opportunities and Challenges for In Vivo CRISPR/Cas-Based Genome Editing

(2020) Tuo Wei et al.   ACS Nano

Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

(2020) Andrew V. Anzalone et al.   NATURE BIOTECHNOLOGY

Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing

(2020) Tuo Wei et al.   Nature Communications

Engineering precision nanoparticles for drug delivery

(2020) Michael J. Mitchell et al.   NATURE REVIEWS DRUG DISCOVERY

Branched-Tail Lipid Nanoparticles Potently Deliver mRNA In Vivo due to Enhanced Ionization at Endosomal pH

(2019) Khalid A. Hajj et al.   Small

CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

(2019) Yi-Li Min et al.   Science Advances

The next generation of CRISPR–Cas technologies and applications

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

Stimulation of CRISPR-mediated homology-directed repair by an engineered RAD18 variant

(2019) Tarun S. Nambiar et al.   Nature Communications

Rational designs of in vivo CRISPR-Cas delivery systems

(2019) Cong-Fei Xu et al.   ADVANCED DRUG DELIVERY REVIEWS

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

Polymer-stabilized Cas9 nanoparticles and modified repair templates increase genome editing efficiency

(2019) David N. Nguyen et al.   NATURE BIOTECHNOLOGY

Advances in genome editing through control of DNA repair pathways

(2019) Charles D. Yeh et al.   NATURE CELL BIOLOGY

Strategies for the CRISPR-Based Therapeutics

(2019) Bin Li et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Development of Cationic Quaternary Ammonium Sulfonamide Amino Lipids for Nucleic Acid Delivery

(2018) Jason B. Miller et al.   ACS Applied Materials & Interfaces

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

(2018) Ling Li et al.   BIOMATERIALS

The Polyploid State Plays a Tumor-Suppressive Role in the Liver

(2018) Shuyuan Zhang et al.   DEVELOPMENTAL CELL

Knockdown of Anillin Actin Binding Protein Blocks Cytokinesis in Hepatocytes and Reduces Liver Tumor Development in Mice Without Affecting Regeneration

(2018) Shuyuan Zhang et al.   GASTROENTEROLOGY

Lipid Nanoparticle Formulations for Enhanced Co-delivery of siRNA and mRNA

(2018) Rebecca L. Ball et al.   NANO LETTERS

Engineering the Delivery System for CRISPR-Based Genome Editing

(2018) Zachary Glass et al.   TRENDS IN BIOTECHNOLOGY

Design of synthetic materials for intracellular delivery of RNAs: From siRNA-mediated gene silencing to CRISPR/Cas gene editing

(2018) Jason B. Miller et al.   Nano Research

CRISPR-Cas guides the future of genetic engineering

(2018) Gavin J. Knott et al.   SCIENCE

Dendrimer-Based Lipid Nanoparticles Deliver Therapeutic FAH mRNA to Normalize Liver Function and Extend Survival in a Mouse Model of Hepatorenal Tyrosinemia Type I

(2018) Qiang Cheng et al.   ADVANCED MATERIALS

CRISPR Correction of Duchenne Muscular Dystrophy

(2018) Yi-Li Min et al.   Annual Review of Medicine

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

(2018) Annalisa Lattanzi et al.   MOLECULAR THERAPY

Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models

(2017) Randy J. Chandler et al.   HUMAN GENE THERAPY

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

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

Boosting Intracellular Delivery of Lipid Nanoparticle-Encapsulated mRNA

(2017) Siddharth Patel et al.   NANO LETTERS

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

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

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

The democratization of gene editing: Insights from site-specific cleavage and double-strand break repair

(2016) Maria Jasin et al.   DNA REPAIR

Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA

(2016) Christopher D Richardson 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

Functional polyesters enable selective siRNA delivery to lung cancer over matched normal cells

(2016) Yunfeng Yan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Modular degradable dendrimers enable small RNAs to extend survival in an aggressive liver cancer model

(2016) Kejin Zhou et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

RS-1 enhances CRISPR/Cas9- and TALEN-mediated knock-in efficiency

(2016) Jun Song et al.   Nature Communications

Post-translational Regulation of Cas9 during G1 Enhances Homology-Directed Repair

(2016) Tony Gutschner et al.   Cell Reports

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy

(2015) Randy J. Chandler et al.   JOURNAL OF CLINICAL INVESTIGATION

Rapid Synthesis of a Lipocationic Polyester Library via Ring-Opening Polymerization of Functional Valerolactones for Efficacious siRNA Delivery

(2015) Jing Hao et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo

(2015) Bin Li et al.   NANO LETTERS

Optimization of Lipid Nanoparticle Formulations for mRNA Delivery in Vivo with Fractional Factorial and Definitive Screening Designs

(2015) Kevin J. Kauffman et al.   NANO LETTERS

Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown

(2015) Anders Wittrup et al.   NATURE BIOTECHNOLOGY

Nuclear domain ‘knock-in’ screen for the evaluation and identification of small molecule enhancers of CRISPR-based genome editing

(2015) Jordan Pinder et al.   NUCLEIC ACIDS RESEARCH

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

(2015) C. Long et al.   SCIENCE

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling

(2014) Randall J. Platt et al.   CELL

Rapid modelling of cooperating genetic events in cancer through somatic genome editing

(2014) Francisco J. Sánchez-Rivera et al.   NATURE

CRISPR-Cas systems for editing, regulating and targeting genomes

(2014) Jeffry D Sander et al.   NATURE BIOTECHNOLOGY

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

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

(2014) Steven Lin et al.   eLife

Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape

(2013) Jerome Gilleron et al.   NATURE BIOTECHNOLOGY

Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recycling

(2013) Gaurav Sahay et al.   NATURE BIOTECHNOLOGY

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

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

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

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

(2011) Elitza Deltcheva et al.   NATURE

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

(2010) Josiane E. Garneau et al.   NATURE

Short motif sequences determine the targets of the prokaryotic CRISPR defence system

(2009)   MICROBIOLOGY-SGM