Strategies for nonviral nanoparticle‐based delivery of CRISPR/Cas9 therapeutics

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

Tumor immune microenvironment and nano-immunotherapeutics in colorectal cancer

(2019) Yang Xiong et al.   Nanomedicine-Nanotechnology Biology and Medicine

Nanoparticle-Mediated Trapping of Wnt Family Member 5A in Tumor Microenvironments Enhances Immunotherapy for B-Raf Proto-Oncogene Mutant Melanoma

(2018) Qi Liu et al.   ACS Nano

Nanocarrier-Mediated Chemo-Immuno Therapy Arrested Cancer Progression and Induced Tumor Dormancy in Desmoplastic Melanoma

(2018) Qi Liu et al.   ACS Nano

Targeted drug delivery to melanoma

(2018) Qi Liu et al.   ADVANCED DRUG DELIVERY REVIEWS

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

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

Reduced Blood Lipid Levels With In Vivo CRISPR-Cas9 Base Editing of ANGPTL3

(2018) Alexandra C. Chadwick et al.   CIRCULATION

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

(2018) Zhen Zhang et al.   MACROMOLECULAR RAPID COMMUNICATIONS

Effective PEI-mediated delivery of CRISPR-Cas9 complex for targeted gene therapy

(2018) Nari Ryu et al.   Nanomedicine-Nanotechnology Biology and Medicine

Engineering the Delivery System for CRISPR-Based Genome Editing

(2018) Zachary Glass et al.   TRENDS IN BIOTECHNOLOGY

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

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

Systemic delivery of CRISPR/Cas9 with PEG-PLGA nanoparticles for chronic myeloid leukemia targeted therapy

(2018) Yang Liu et al.   Biomaterials 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

PEGylated Chitosan for Non-Viral Aerosol and Mucosal Delivery of CRISPR-Cas9 System in Vitro

(2018) Hairui Zhang et al.   MOLECULAR PHARMACEUTICS

Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics

(2018) Brittany E. Givens et al.   AAPS Journal

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

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

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

(2017) Hao Yin et al.   NATURE BIOTECHNOLOGY

Advances in the delivery of RNA therapeutics: from concept to clinical reality

(2017) James C. Kaczmarek et al.   Genome Medicine

Nanoparticles for CRISPR–Cas9 delivery

(2017) Zachary Glass et al.   Nature Biomedical Engineering

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

Polymer-Lipid Nanoparticles for Systemic Delivery of mRNA to the Lungs

(2016) James C. Kaczmarek et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Membrane Microdomain Structures of Liposomes and Their Contribution to the Cellular Uptake Efficiency into HeLa Cells

(2016) Yoshinori Onuki et al.   MOLECULAR PHARMACEUTICS

Engineered Viruses as Genome Editing Devices

(2016) Xiaoyu Chen et al.   MOLECULAR THERAPY

Chinese scientists to pioneer first human CRISPR trial

(2016) David Cyranoski   NATURE

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

(2016) Daniel P. Dever et al.   NATURE

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

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

Editing the epigenome: technologies for programmable transcription and epigenetic modulation

(2016) Pratiksha I Thakore et al.   NATURE METHODS

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles

(2016) Ming Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Targeted delivery of CRISPR/Cas9 to prostate cancer by modified gRNA using a flexible aptamer-cationic liposome

(2016) Shuai Zhen et al.   Oncotarget

A CRISPR Dropout Screen Identifies Genetic Vulnerabilities and Therapeutic Targets in Acute Myeloid Leukemia

(2016) Konstantinos Tzelepis et al.   Cell Reports

Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids

(2016) Kyle Chamberlain et al.   Human Gene Therapy Methods

Glycyrrhetinic acid-decorated and reduction-sensitive micelles to enhance the bioavailability and anti-hepatocellular carcinoma efficacy of tanshinone IIA

(2016) Fengqian Chen et al.   Biomaterials Science

Intracellular delivery of the PTEN protein using cationic lipidoids for cancer therapy

(2016) Sarah A. Altınoğlu et al.   Biomaterials Science

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

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

Targeting hepatitis B virus cccDNA by CRISPR/Cas9 nuclease efficiently inhibits viral replication

(2015) Chunsheng Dong et al.   ANTIVIRAL RESEARCH

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

(2015) Florian Schmidt et al.   Biotechnology Journal

Cancer-Associated Protein Kinase C Mutations Reveal Kinase’s Role as Tumor Suppressor

(2015) Corina E. Antal et al.   CELL

Engineering Complex Synthetic Transcriptional Programs with CRISPR RNA Scaffolds

(2015) Jesse G. Zalatan et al.   CELL

Harnessing the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated Cas9 system to disrupt the hepatitis B virus

(2015) S Zhen et al.   GENE THERAPY

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

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

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

(2015) Ling Li et al.   HUMAN GENE THERAPY

Polymeric Nanocarriers for Non-Viral Gene Delivery

(2015) Ling Li et al.   Journal of Biomedical Nanotechnology

Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection

(2015) Xiquan Liang et al.   JOURNAL OF BIOTECHNOLOGY

Nucleolin targeting AS1411 aptamer modified pH-sensitive micelles for enhanced delivery and antitumor efficacy of paclitaxel

(2015) Jinming Zhang et al.   Nano Research

Combinatorial library strategies for synthesis of cationic lipid-like nanoparticles and their potential medical applications

(2015) Sarah Altınoglu et al.   Nanomedicine

Tumor pHe-triggered charge-reversal and redox-responsive nanoparticles for docetaxel delivery in hepatocellular carcinoma treatment

(2015) Fengqian Chen et al.   Nanoscale

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

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

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

Cas9-mediated targeting of viral RNA in eukaryotic cells

(2015) Aryn A. Price 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

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

(2015) Marc Zuckermann et al.   Nature Communications

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

(2015) Florian Schmidt et al.   Biotechnology Journal

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

(2014) Elena Senís et al.   Biotechnology Journal

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

Preparation, characterization, and anticancer efficacy of evodiamine-loaded PLGA nanoparticles

(2014) Lidi Zou et al.   DRUG DELIVERY

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

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

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

CRISPR–Cas systems: beyond adaptive immunity

(2014) Edze R. Westra et al.   NATURE REVIEWS MICROBIOLOGY

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Synthesizing AND gate genetic circuits based on CRISPR-Cas9 for identification of bladder cancer cells

(2014) Yuchen Liu et al.   Nature Communications

Genetics and genomics of Parkinson’s disease

(2014) Michelle K Lin et al.   Genome Medicine

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

(2014) Steven Lin et al.   eLife

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

(2014) Syandan Chakraborty et al.   Stem Cell Reports

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

(2013) Lei S. Qi et al.   CELL

One-Step Generation of Mice Carrying Reporter and Conditional Alleles by CRISPR/Cas-Mediated Genome Engineering

(2013) Hui Yang et al.   CELL

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

(2013) F. Ann Ran et al.   CELL

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

(2013) Luke A. Gilbert et al.   CELL

Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

(2013) Gerald Schwank et al.   Cell Stem Cell

CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering

(2013) Prashant Mali et al.   NATURE BIOTECHNOLOGY

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Programmable repression and activation of bacterial gene expression using an engineered CRISPR-Cas system

(2013) David Bikard et al.   NUCLEIC ACIDS RESEARCH

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

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

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

(2013) Hirotaka Ebina et al.   Scientific Reports

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

(2012) M. Jinek et al.   SCIENCE

Strategies for extended serum half-life of protein therapeutics

(2011) Roland E Kontermann   CURRENT OPINION IN BIOTECHNOLOGY

Cholesterol-Dependent Macropinocytosis and Endosomal Escape Control the Transfection Efficiency of Lipoplexes in CHO Living Cells

(2011) Francesco Cardarelli et al.   MOLECULAR PHARMACEUTICS

Accumulation of sub-100 nm polymeric micelles in poorly permeable tumours depends on size

(2011) H. Cabral et al.   Nature Nanotechnology

State-of-the-art gene-based therapies: the road ahead

(2011) Mark A. Kay   NATURE REVIEWS GENETICS

Exploring the Genomes of Cancer Cells: Progress and Promise

(2011) M. R. Stratton   SCIENCE

Poly(ethylene glycol) in Drug Delivery: Pros and Cons as Well as Potential Alternatives

(2010) Katrin Knop et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Polymer- and lipid-based nanoparticle therapeutics for the treatment of liver diseases

(2010) Lanjuan Li et al.   Nano Today

Knocking down barriers: advances in siRNA delivery

(2009) Kathryn A. Whitehead et al.   NATURE REVIEWS DRUG DISCOVERY

Nonviral Vectors for Gene Delivery

(2008) Meredith A. Mintzer et al.   CHEMICAL REVIEWS

Factors Affecting the Clearance and Biodistribution of Polymeric Nanoparticles

(2008) Frank Alexis et al.   MOLECULAR PHARMACEUTICS

Thirty years of Alzheimer's disease genetics: the implications of systematic meta-analyses

(2008) Lars Bertram et al.   NATURE REVIEWS NEUROSCIENCE

Cationic liposomal lipids: From gene carriers to cell signaling

(2008) Caroline Lonez et al.   PROGRESS IN LIPID RESEARCH

Small CRISPR RNAs Guide Antiviral Defense in Prokaryotes

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