Co-encapsulation of Cas9 mRNA and guide RNA in polyplex micelles enables genome editing in mouse brain

The promise and challenge of therapeutic genome editing

(2020) Jennifer A. Doudna   NATURE

Glia-to-Neuron Conversion by CRISPR-CasRx Alleviates Symptoms of Neurological Disease in Mice

(2020) Haibo Zhou et al.   CELL

Self‐Degradable Lipid‐Like Materials Based on “Hydrolysis accelerated by the intra‐Particle Enrichment of Reactant (HyPER)” for Messenger RNA Delivery

(2020) Hiroki Tanaka et al.   ADVANCED FUNCTIONAL MATERIALS

Reversing a model of Parkinson’s disease with in situ converted nigral neurons

(2020) Hao Qian et al.   NATURE

Design concepts of polyplex micelles for in vivo therapeutic delivery of plasmid DNA and messenger RNA

(2019) Satoshi Uchida et al.   JOURNAL OF BIOMEDICAL MATERIALS RESEARCH PART A

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

(2019) Morgan L. Maeder et al.   NATURE MEDICINE

In vivo neuronal gene editing via CRISPR–Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer’s disease

(2019) Hanseul Park et al.   NATURE NEUROSCIENCE

Identification of preexisting adaptive immunity to Cas9 proteins in humans

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

Genome Editing with mRNA Encoding ZFN, TALEN, and Cas9

(2019) Hong-Xia Zhang et al.   MOLECULAR THERAPY

Induced packaging of mRNA into polyplex micelles by regulated hybridization with a small number of cholesteryl RNA oligonucleotides directed enhanced in vivo transfection

(2019) Naoto Yoshinaga et al.   BIOMATERIALS

Enhancement of Motor Function Recovery after Spinal Cord Injury in Mice by Delivery of Brain-Derived Neurotrophic Factor mRNA

(2019) Samuel T. Crowley et al.   Molecular Therapy-Nucleic Acids

Fine-Tuning of Hydrophobicity in Amphiphilic Polyaspartamide Derivatives for Rapid and Transient Expression of Messenger RNA Directed Toward Genome Engineering in Brain

(2019) Hyun Jin Kim et al.   ACS Central Science

Block Copolymer Micelles in Nanomedicine Applications

(2018) Horacio Cabral et al.   CHEMICAL REVIEWS

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

Receptor-Mediated Delivery of CRISPR-Cas9 Endonuclease for Cell-Type-Specific Gene Editing

(2018) Romain Rouet et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

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

CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington’s disease

(2017) Su Yang et al.   JOURNAL OF CLINICAL INVESTIGATION

Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes

(2017) Brett T Staahl et al.   NATURE BIOTECHNOLOGY

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

(2017) Hao Yin et al.   NATURE BIOTECHNOLOGY

Polyion complex micelle formation from double-hydrophilic block copolymers composed of charged and non-charged segments in aqueous media

(2017) Atsushi Harada et al.   POLYMER JOURNAL

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

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Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

(2016) Michaël Hocquemiller et al.   HUMAN GENE THERAPY

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

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

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

(2015) C. Long et al.   SCIENCE

RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection

(2014) W. Hu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Convection-Enhanced Drug Delivery to the Brain: Therapeutic Potential and Neuropathological Considerations

(2013) Neil U. Barua et al.   BRAIN PATHOLOGY

Block/homo polyplex micelle-based GM-CSF gene therapy via intraperitoneal administration elicits antitumor immunity against peritoneal dissemination and exhibits safety potentials in mice and cynomolgus monkeys

(2013) Masahiro Ohgidani et al.   JOURNAL OF CONTROLLED RELEASE

In Vivo Messenger RNA Introduction into the Central Nervous System Using Polyplex Nanomicelle

(2013) Satoshi Uchida et al.   PLoS One

PEGylated Polyplex With Optimized PEG Shielding Enhances Gene Introduction in Lungs by Minimizing Inflammatory Responses

(2012) Satoshi Uchida et al.   MOLECULAR THERAPY

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

(2012) M. Jinek et al.   SCIENCE

Enhanced stability and gene silencing ability of siRNA-loaded polyion complexes formulated from polyaspartamide derivatives with a repetitive array of amino groups in the side chain

(2011) Tomoya Suma et al.   BIOMATERIALS

Rational design of smart supramolecular assemblies for gene delivery: chemical challenges in the creation of artificial viruses

(2011) Kanjiro Miyata et al.   CHEMICAL SOCIETY REVIEWS

Biodegradable polyamino acid-based polycations as safe and effective gene carrier minimizing cumulative toxicity

(2010) Keiji Itaka et al.   BIOMATERIALS

Self-Assembled Lipid Nanomedicines for siRNA Tumor Targeting

(2010) Yu-Cheng Tseng et al.   Journal of Biomedical Nanotechnology

Enhanced Percolation and Gene Expression in Tumor Hypoxia by PEGylated Polyplex Micelles

(2009) Muri Han et al.   MOLECULAR THERAPY

A robust and high-throughput Cre reporting and characterization system for the whole mouse brain

(2009) Linda Madisen et al.   NATURE NEUROSCIENCE

Attenuation of Zinc Finger Nuclease Toxicity by Small-Molecule Regulation of Protein Levels

(2009) Shondra M. Pruett-Miller et al.   PLoS Genetics

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