Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo.

PubWeight™: 2.75‹?› | Rank: Top 1%

🔗 View Article (PMC 4289409)

Published in Nat Biotechnol on October 30, 2014

Authors

John A Zuris1, David B Thompson1, Yilai Shu2, John P Guilinger1, Jeffrey L Bessen1, Johnny H Hu1, Morgan L Maeder3, J Keith Joung3, Zheng-Yi Chen4, David R Liu1

Author Affiliations

1: 1] Department of Chemistry &Chemical Biology, Harvard University, Cambridge, Massachusetts, USA. [2] Howard Hughes Medical Institute, Harvard University, Cambridge, Massachusetts, USA.
2: 1] Department of Otology and Laryngology, Harvard Medical School, Boston, Massachusetts, USA. [2] Eaton-Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts, USA. [3] Department of Otology and Skull Base Surgery, Eye, Ear, Nose and Throat Hospital, Shanghai Medical College, Fudan University, Shanghai, China. [4] Key Laboratory of Health Ministry for Hearing Medicine, Shanghai, China.
3: 1] Molecular Pathology Unit, Massachusetts General Hospital, Charlestown, Massachusetts, USA. [2] Center for Cancer Research, Massachusetts General Hospital, Charlestown, Massachusetts, USA. [3] Center for Computational and Integrative Biology, Massachusetts General Hospital, Charlestown, Massachusetts, USA. [4] Department of Pathology, Harvard Medical School, Boston, Massachusetts, USA.
4: 1] Department of Otology and Laryngology, Harvard Medical School, Boston, Massachusetts, USA. [2] Eaton-Peabody Laboratory, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts, USA.

Articles citing this

(truncated to the top 100)

Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery. Elife (2014) 2.65

Therapeutic genome editing: prospects and challenges. Nat Med (2015) 2.42

Chemical tagging and customizing of cellular chromatin states using ultrafast trans-splicing inteins. Nat Chem (2015) 2.17

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature (2016) 2.15

Small molecule-triggered Cas9 protein with improved genome-editing specificity. Nat Chem Biol (2015) 2.13

Programmable RNA Tracking in Live Cells with CRISPR/Cas9. Cell (2016) 1.75

Cloning-free CRISPR/Cas system facilitates functional cassette knock-in in mice. Genome Biol (2015) 1.64

Generation of knock-in primary human T cells using Cas9 ribonucleoproteins. Proc Natl Acad Sci U S A (2015) 1.49

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo. Nat Biotechnol (2016) 1.44

Dependency of a therapy-resistant state of cancer cells on a lipid peroxidase pathway. Nature (2017) 1.42

Targeted Mutagenesis, Precise Gene Editing, and Site-Specific Gene Insertion in Maize Using Cas9 and Guide RNA. Plant Physiol (2015) 1.39

Applications of CRISPR technologies in research and beyond. Nat Biotechnol (2016) 1.34

Applications of the CRISPR-Cas9 system in cancer biology. Nat Rev Cancer (2015) 1.32

Photoactivatable CRISPR-Cas9 for optogenetic genome editing. Nat Biotechnol (2015) 1.28

Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses. Hum Gene Ther (2015) 1.18

Applications of CRISPR-Cas systems in neuroscience. Nat Rev Neurosci (2015) 1.18

A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear. Nat Biotechnol (2017) 1.14

Advances in CRISPR-Cas9 genome engineering: lessons learned from RNA interference. Nucleic Acids Res (2015) 1.10

CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes. Cell (2016) 1.08

Efficient and transgene-free genome editing in wheat through transient expression of CRISPR/Cas9 DNA or RNA. Nat Commun (2016) 1.04

Defining and improving the genome-wide specificities of CRISPR-Cas9 nucleases. Nat Rev Genet (2016) 1.03

Genome-wide target specificities of CRISPR-Cas9 nucleases revealed by multiplex Digenome-seq. Genome Res (2016) 1.01

Synthetic epigenetics-towards intelligent control of epigenetic states and cell identity. Clin Epigenetics (2015) 0.99

Measuring and Reducing Off-Target Activities of Programmable Nucleases Including CRISPR-Cas9. Mol Cells (2015) 0.97

Creating and evaluating accurate CRISPR-Cas9 scalpels for genomic surgery. Nat Methods (2015) 0.96

Resources for the design of CRISPR gene editing experiments. Genome Biol (2015) 0.95

Large genomic fragment deletion and functional gene cassette knock-in via Cas9 protein mediated genome editing in one-cell rodent embryos. Sci Rep (2015) 0.93

In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa. Mol Ther (2015) 0.92

Self-Assembled DNA Nanoclews for the Efficient Delivery of CRISPR-Cas9 for Genome Editing. Angew Chem Int Ed Engl (2015) 0.91

Methods for Optimizing CRISPR-Cas9 Genome Editing Specificity. Mol Cell (2016) 0.91

Enabling functional genomics with genome engineering. Genome Res (2015) 0.90

Homology-directed repair in rodent zygotes using Cas9 and TALEN engineered proteins. Sci Rep (2015) 0.90

DNA-free two-gene knockout in Chlamydomonas reinhardtii via CRISPR-Cas9 ribonucleoproteins. Sci Rep (2016) 0.90

Precision cancer mouse models through genome editing with CRISPR-Cas9. Genome Med (2015) 0.89

DNA-binding-domain fusions enhance the targeting range and precision of Cas9. Nat Methods (2015) 0.88

Structure and Engineering of Francisella novicida Cas9. Cell (2016) 0.88

Continuous directed evolution of DNA-binding proteins to improve TALEN specificity. Nat Methods (2015) 0.88

Genome Editing in Human Pluripotent Stem Cells: Approaches, Pitfalls, and Solutions. Cell Stem Cell (2016) 0.87

Modeling Disease In Vivo With CRISPR/Cas9. Trends Mol Med (2015) 0.87

Genome-editing technologies for gene correction of hemophilia. Hum Genet (2016) 0.85

Applications of Cas9 as an RNA-programmed RNA-binding protein. Bioessays (2015) 0.84

CRISPR/Cas9-induced disruption of gene expression in mouse embryonic brain and single neural stem cells in vivo. EMBO Rep (2016) 0.83

A new age in functional genomics using CRISPR/Cas9 in arrayed library screening. Front Genet (2015) 0.83

Intracellular Delivery of Proteins via Fusion Peptides in Intact Plants. PLoS One (2016) 0.83

CRISPR: a versatile tool for both forward and reverse genetics research. Hum Genet (2016) 0.82

The application of genome editing in studying hearing loss. Hear Res (2015) 0.82

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles. Proc Natl Acad Sci U S A (2016) 0.81

Genome editing comes of age. Nat Protoc (2016) 0.81

Advances in Nano-based inner ear delivery systems for the treatment of sensorineural hearing loss. Adv Drug Deliv Rev (2016) 0.81

Treating hemoglobinopathies using gene-correction approaches: promises and challenges. Hum Genet (2016) 0.81

Different Effects of sgRNA Length on CRISPR-mediated Gene Knockout Efficiency. Sci Rep (2016) 0.81

A de novo silencer causes elimination of MITF-M expression and profound hearing loss in pigs. BMC Biol (2016) 0.80

Improved cell-penetrating zinc-finger nuclease proteins for precision genome engineering. Mol Ther Nucleic Acids (2015) 0.80

CRISPR/Cas9-Mediated Genome Editing of Herpesviruses Limits Productive and Latent Infections. PLoS Pathog (2016) 0.80

Multidimensional chemical control of CRISPR-Cas9. Nat Chem Biol (2016) 0.80

In Vivo Excision of HIV-1 Provirus by saCas9 and Multiplex Single-Guide RNAs in Animal Models. Mol Ther (2017) 0.79

Combining CRISPR/Cas9 and rAAV Templates for Efficient Gene Editing. Nucleic Acid Ther (2015) 0.79

Protein Delivery of an Artificial Transcription Factor Restores Widespread Ube3a Expression in an Angelman Syndrome Mouse Brain. Mol Ther (2016) 0.79

Strategies for precision modulation of gene expression by epigenome editing: an overview. Epigenetics Chromatin (2015) 0.79

High-Content Analysis of CRISPR-Cas9 Gene-Edited Human Embryonic Stem Cells. Stem Cell Reports (2016) 0.79

Structurally Programmed Assembly of Translation Initiation Nanoplex for Superior mRNA Delivery. ACS Nano (2017) 0.79

The Rise of CRISPR/Cas for Genome Editing in Stem Cells. Stem Cells Int (2016) 0.78

Identification of Adeno-Associated Viral Vectors That Target Neonatal and Adult Mammalian Inner Ear Cell Subtypes. Hum Gene Ther (2016) 0.78

The history and market impact of CRISPR RNA-guided nucleases. Curr Opin Virol (2015) 0.78

Salient Features of Endonuclease Platforms for Therapeutic Genome Editing. Mol Ther (2016) 0.78

Controlled delivery of β-globin-targeting TALENs and CRISPR/Cas9 into mammalian cells for genome editing using microinjection. Sci Rep (2015) 0.78

Genome surgery using Cas9 ribonucleoproteins for the treatment of age-related macular degeneration. Genome Res (2017) 0.78

Cell-Penetrating Peptide as a Means of Directing the Differentiation of Induced-Pluripotent Stem Cells. Int J Mol Sci (2015) 0.77

Non-viral delivery of genome-editing nucleases for gene therapy. Gene Ther (2016) 0.77

Oscillating high-aspect-ratio monolithic silicon nanoneedle array enables efficient delivery of functional bio-macromolecules into living cells. Sci Rep (2015) 0.77

A genome editing primer for the hematologist. Blood (2016) 0.77

High content analysis platform for optimization of lipid mediated CRISPR-Cas9 delivery strategies in human cells. Acta Biomater (2015) 0.77

The CRISPR revolution and its impact on cancer research. Swiss Med Wkly (2015) 0.77

Improved delivery of Cas9 protein/gRNA complexes using lipofectamine CRISPRMAX. Biotechnol Lett (2016) 0.76

Developmental regulation of planar cell polarity and hair-bundle morphogenesis in auditory hair cells: lessons from human and mouse genetics. Wiley Interdiscip Rev Dev Biol (2015) 0.76

Intracellular targeting with engineered proteins. F1000Res (2016) 0.76

Engineering Strategies to Decode and Enhance the Genomes of Coral Symbionts. Front Microbiol (2017) 0.76

Genome Engineering with TALE and CRISPR Systems in Neuroscience. Front Genet (2016) 0.75

Affinity-controlled protein encapsulation into sub-30 nm telodendrimer nanocarriers by multivalent and synergistic interactions. Biomaterials (2016) 0.75

Scratching the Surface: Resurfacing Proteins to Endow New Properties and Function. Cell Chem Biol (2016) 0.75

Lipid-based Transfection Reagents Exhibit Cryo-induced Increase in Transfection Efficiency. Mol Ther Nucleic Acids (2016) 0.75

Overcoming doxorubicin resistance of cancer cells by Cas9-mediated gene disruption. Sci Rep (2016) 0.75

Genomic targeting of epigenetic probes using a chemically tailored Cas9 system. Proc Natl Acad Sci U S A (2017) 0.75

In Vivo Delivery Systems for Therapeutic Genome Editing. Int J Mol Sci (2016) 0.75

Prospects and challenges of CRISPR/Cas genome editing for the study and control of neglected vector-borne nematode diseases. FEBS J (2016) 0.75

Cytosolic delivery of large proteins using nanoparticle-stabilized nanocapsules. Nanoscale (2016) 0.75

Survival and Evolution of CRISPR-Cas System in Prokaryotes and Its Applications. Front Immunol (2016) 0.75

Cre Fused with RVG Peptide Mediates Targeted Genome Editing in Mouse Brain Cells In Vivo. Int J Mol Sci (2016) 0.75

CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10. Mol Ther (2017) 0.75

Cochlear hair cell regeneration after noise-induced hearing loss: Does regeneration follow development? Hear Res (2016) 0.75

Hearing Assessment in Zebrafish During the First Week Postfertilization. Zebrafish (2016) 0.75

Comprehensive Protocols for CRISPR/Cas9-based Gene Editing in Human Pluripotent Stem Cells. Curr Protoc Stem Cell Biol (2016) 0.75

Sendai virus, an RNA virus with no risk of genomic integration, delivers CRISPR/Cas9 for efficient gene editing. Mol Ther Methods Clin Dev (2016) 0.75

Perspectives of Genome-Editing Technologies for HIV Therapy. Curr HIV Res (2016) 0.75

Brain tumor modeling using the CRISPR/Cas9 system: state of the art and view to the future. Oncotarget (2016) 0.75

CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells. Korean J Intern Med (2017) 0.75

CRISPR/Cas9 therapeutics: a cure for cancer and other genetic diseases. Oncotarget (2016) 0.75

Application of the genome editing tool CRISPR/Cas9 in non-human primates. Dongwuxue Yanjiu (2016) 0.75

CRISPR-Mediated Epigenome Editing. Yale J Biol Med (2016) 0.75

Protein transduction therapy into cochleae via the round window niche in guinea pigs. Mol Ther Methods Clin Dev (2016) 0.75

Articles cited by this

NIH Image to ImageJ: 25 years of image analysis. Nat Methods (2012) 84.41

RNA-guided human genome engineering via Cas9. Science (2013) 48.29

One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering. Cell (2013) 24.00

Genome editing with engineered zinc finger nucleases. Nat Rev Genet (2010) 18.88

Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression. Cell (2013) 13.43

Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity. Cell (2013) 13.37

CRISPR-Cas systems for editing, regulating and targeting genomes. Nat Biotechnol (2014) 9.18

High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity. Nat Biotechnol (2013) 8.40

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs. Nat Biotechnol (2014) 6.85

A rapid and general assay for monitoring endogenous gene modification. Methods Mol Biol (2010) 6.77

Transducible TAT-HA fusogenic peptide enhances escape of TAT-fusion proteins after lipid raft macropinocytosis. Nat Med (2004) 6.48

Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors. Nat Biotechnol (2005) 6.40

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nat Biotechnol (2014) 5.71

CRISPR RNA-guided activation of endogenous human genes. Nat Methods (2013) 4.69

Protein therapeutics: a summary and pharmacological classification. Nat Rev Drug Discov (2008) 4.32

Liposomal drug delivery systems: from concept to clinical applications. Adv Drug Deliv Rev (2012) 4.30

Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat Med (2001) 4.02

Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med (2013) 3.97

A peptide carrier for the delivery of biologically active proteins into mammalian cells. Nat Biotechnol (2001) 3.86

Fusion of catalytically inactive Cas9 to FokI nuclease improves the specificity of genome modification. Nat Biotechnol (2014) 3.72

Math1-driven GFP expression in the developing nervous system of transgenic mice. Gene Expr Patterns (2003) 3.42

Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins. Genome Res (2014) 3.31

Design of noninflammatory synthetic siRNA mediating potent gene silencing in vivo. Mol Ther (2005) 3.28

Robust, synergistic regulation of human gene expression using TALE activators. Nat Methods (2013) 2.89

Proliferation of functional hair cells in vivo in the absence of the retinoblastoma protein. Science (2005) 2.81

Image-based analysis of lipid nanoparticle-mediated siRNA delivery, intracellular trafficking and endosomal escape. Nat Biotechnol (2013) 2.81

Supercharging proteins can impart unusual resilience. J Am Chem Soc (2007) 2.52

Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction. Nat Biotechnol (2013) 2.11

Mammalian cell penetration, siRNA transfection, and DNA transfection by supercharged proteins. Proc Natl Acad Sci U S A (2009) 1.95

Gene disruption by cell-penetrating peptide-mediated delivery of Cas9 protein and guide RNA. Genome Res (2014) 1.91

Systemic in vivo distribution of activatable cell penetrating peptides is superior to that of cell penetrating peptides. Integr Biol (Camb) (2009) 1.78

Chemical vectors for gene delivery: a current review on polymers, peptides and lipids containing histidine or imidazole as nucleic acids carriers. Br J Pharmacol (2009) 1.77

Improving protein therapeutics with sustained-release formulations. Nat Biotechnol (1998) 1.73

Electroporation for the delivery of DNA-based vaccines and immunotherapeutics: current clinical developments. Mol Ther (2009) 1.69

Intracellular delivery of proteins with a new lipid-mediated delivery system. J Biol Chem (2001) 1.65

Targeted mutation in the neurotrophin-3 gene results in loss of muscle sensory neurons. Proc Natl Acad Sci U S A (1994) 1.54

Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment. Nat Biotechnol (2008) 1.50

In silico abstraction of zinc finger nuclease cleavage profiles reveals an expanded landscape of off-target sites. Nucleic Acids Res (2013) 1.46

Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene. Mol Ther (2004) 1.44

Potent delivery of functional proteins into Mammalian cells in vitro and in vivo using a supercharged protein. ACS Chem Biol (2010) 1.42

Protein encapsulation in liposomes: efficiency depends on interactions between protein and phospholipid bilayer. BMC Biotechnol (2002) 1.29

Intrinsically cell-permeable miniature proteins based on a minimal cationic PPII motif. J Am Chem Soc (2007) 1.27

Cell-penetrating peptide-mediated delivery of TALEN proteins via bioconjugation for genome engineering. PLoS One (2014) 1.24

Influence of cationic lipid composition on gene silencing properties of lipid nanoparticle formulations of siRNA in antigen-presenting cells. Mol Ther (2011) 1.23

Engineering and identifying supercharged proteins for macromolecule delivery into mammalian cells. Methods Enzymol (2012) 0.97

Endosomal escape: a bottleneck in intracellular delivery. J Nanosci Nanotechnol (2014) 0.96

Cellular uptake mechanisms and endosomal trafficking of supercharged proteins. Chem Biol (2012) 0.95

Targeted SAINT-O-Somes for improved intracellular delivery of siRNA and cytotoxic drugs into endothelial cells. J Control Release (2010) 0.86

Latent cytokines for targeted therapy of inflammatory disorders. Expert Opin Drug Deliv (2013) 0.80

Articles by these authors

Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing. Nat Biotechnol (2014) 4.41