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

Improved delivery of Cas9 protein/gRNA complexes using lipofectamine CRISPRMAX

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CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice

(2016) Li Xu et al.   MOLECULAR THERAPY

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

(2016) Daniel P. Dever et al.   NATURE

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

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

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

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

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

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

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

Spherical Nucleic Acids as a Divergent Platform for Synthesizing RNA–Nanoparticle Conjugates through Enzymatic Ligation

(2014) Jessica L. Rouge et al.   ACS Nano

Protein Corona Fingerprinting Predicts the Cellular Interaction of Gold and Silver Nanoparticles

(2014) Carl D. Walkey et al.   ACS Nano

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

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

(2014) S. Kim et al.   GENOME RESEARCH

Gold Nanoparticles for Nucleic Acid Delivery

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DNA interrogation by the CRISPR RNA-guided endonuclease Cas9

(2014) Samuel H. Sternberg 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

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

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

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

DNA assembly of nanoparticle superstructures for controlled biological delivery and elimination

(2014) Leo Y. T. Chou et al.   Nature Nanotechnology

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

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Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

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

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

Optimization of scarless human stem cell genome editing

(2013) Luhan Yang et al.   NUCLEIC ACIDS RESEARCH

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

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

(2012) M. Jinek et al.   SCIENCE

Exon-skipping therapy for Duchenne muscular dystrophy

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Introduction of stearoyl moieties into a biocompatible cationic polyaspartamide derivative, PAsp(DET), with endosomal escaping function for enhanced siRNA-mediated gene knockdown

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Widespread Muscle Expression of an AAV9 Human Mini-dystrophin Vector After Intravenous Injection in Neonatal Dystrophin-deficient Dogs

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Layer-by-Layer Platform Technology for Small-Molecule Delivery

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Microarray-based mutation detection in thedystrophingene

(2008) Madhuri R. Hegde et al.   HUMAN MUTATION

Polyplexes from Poly(aspartamide) Bearing 1,2-Diaminoethane Side Chains Induce pH-Selective, Endosomal Membrane Destabilization with Amplified Transfection and Negligible Cytotoxicity

(2008) Kanjiro Miyata et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Sustained release of a p38 inhibitor from non-inflammatory microspheres inhibits cardiac dysfunction

(2008) Jay C. Sy et al.   NATURE MATERIALS