Blood-brain barrier–penetrating single CRISPR-Cas9 nanocapsules for effective and safe glioblastoma gene therapy

Development of Novel Therapeutics Targeting the Blood–Brain Barrier: From Barrier to Carrier

(2021) Jia Li et al.   Advanced Science

Overcoming blood–brain barrier transport: Advances in nanoparticle-based drug delivery strategies

(2020) Shichao Ding et al.   Materials Today

Single siRNA Nanocapsules for Effective siRNA Brain Delivery and Glioblastoma Treatment

(2020) Yan Zou et al.   ADVANCED MATERIALS

Modeling the complex genetic architectures of brain disease

(2020) Michael B. Fernando et al.   NATURE GENETICS

Gene-edited human stem cell–derived β cells from a patient with monogenic diabetes reverse preexisting diabetes in mice

(2020) Kristina G. Maxwell et al.   Science Translational Medicine

Physiological blood–brain transport is impaired with age by a shift in transcytosis

(2020) Andrew C. Yang et al.   NATURE

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

Deep brain stimulation: current challenges and future directions

(2019) Andres M. Lozano et al.   Nature Reviews Neurology

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

A Rationally Designed Semiconducting Polymer Brush for NIR‐II Imaging‐Guided Light‐Triggered Remote Control of CRISPR/Cas9 Genome Editing

(2019) Ling Li et al.   ADVANCED MATERIALS

CD22 blockade restores homeostatic microglial phagocytosis in ageing brains

(2019) John V. Pluvinage et al.   NATURE

A CRISPR-Cas9 delivery system for in vivo screening of genes in the immune system

(2019) Martin W. LaFleur et al.   Nature Communications

Application of Förster Resonance Energy Transfer (FRET) technique to elucidate intracellular and In Vivo biofate of nanomedicines

(2019) Tongkai Chen et al.   ADVANCED DRUG DELIVERY REVIEWS

Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles

(2019) Ji Liu et al.   ADVANCED MATERIALS

ROS‐Responsive Polymeric siRNA Nanomedicine Stabilized by Triple Interactions for the Robust Glioblastoma Combinational RNAi Therapy

(2019) Meng Zheng et al.   ADVANCED MATERIALS

CRISPR–Cas9 strikes out in cassava

(2019) Edward P. Rybicki   NATURE BIOTECHNOLOGY

CFTR-PTEN–dependent mitochondrial metabolic dysfunction promotes Pseudomonas aeruginosa airway infection

(2019) Sebastián A. Riquelme et al.   Science Translational Medicine

A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing

(2019) Guojun Chen et al.   Nature Nanotechnology

The blood-brain barrier: Physiology and strategies for drug delivery

(2019) Rucha Pandit et al.   ADVANCED DRUG DELIVERY REVIEWS

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

(2018) Ling Li et al.   BIOMATERIALS

CRISPR interference-based specific and efficient gene inactivation in the brain

(2018) Yi Zheng et al.   NATURE NEUROSCIENCE

Gold rush to gene-editing in the brain

(2018) Michelle Trenkmann   NATURE REVIEWS GENETICS

Nonviral gene editing via CRISPR/Cas9 delivery by membrane-disruptive and endosomolytic helical polypeptide

(2018) Hong-Xia Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Nanotechnology-Based Strategies for siRNA Brain Delivery for Disease Therapy

(2018) Meng Zheng et al.   TRENDS IN BIOTECHNOLOGY

Amphiphilic nanocarrier-induced modulation of PLK1 and miR-34a leads to improved therapeutic response in pancreatic cancer

(2018) Hadas Gibori et al.   Nature Communications

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

Reprogramming human T cell function and specificity with non-viral genome targeting

(2018) Theodore L. Roth et al.   NATURE

Mechanisms of enhanced drug delivery in brain metastases with focused ultrasound-induced blood–tumor barrier disruption

(2018) Costas D. Arvanitis et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Non-Viral Delivery To Enable Genome Editing

(2018) Yuan Rui et al.   TRENDS IN BIOTECHNOLOGY

Cas9 immunity creates challenges for CRISPR gene editing therapies

(2018) Julie M. Crudele et al.   Nature Communications

Nano based drug delivery systems: recent developments and future prospects

(2018) Jayanta Kumar Patra et al.   JOURNAL OF NANOBIOTECHNOLOGY

Apolipoprotein E Peptide-Directed Chimeric Polymersomes Mediate an Ultrahigh-Efficiency Targeted Protein Therapy for Glioblastoma

(2018) Yu Jiang et al.   ACS Nano

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

(2017) Brett T Staahl et al.   NATURE BIOTECHNOLOGY

Cytoplasmic p53 couples oncogene-driven glucose metabolism to apoptosis and is a therapeutic target in glioblastoma

(2017) Wilson X Mai et al.   NATURE MEDICINE

Genome Editing for Cancer Therapy: Delivery of Cas9 Protein/sgRNA Plasmid via a Gold Nanocluster/Lipid Core-Shell Nanocarrier

(2017) Peng Wang et al.   Advanced Science

Growth-Factor Nanocapsules That Enable Tunable Controlled Release for Bone Regeneration

(2016) Haijun Tian et al.   ACS Nano

Artificial Virus Delivers CRISPR-Cas9 System for Genome Editing of Cells in Mice

(2016) Ling Li et al.   ACS Nano

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

Non-viral gene therapy: Gains and challenges of non-invasive administration methods

(2016) Marianna Foldvari et al.   JOURNAL OF CONTROLLED RELEASE

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

(2016) Benjamin P. Kleinstiver 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

From blood–brain barrier to blood–brain interface: new opportunities for CNS drug delivery

(2016) William A. Banks   NATURE REVIEWS DRUG DISCOVERY

Progress and challenges in viral vector manufacturing

(2015) Johannes C.M. van der Loo et al.   HUMAN MOLECULAR GENETICS

High-throughput functional genomics using CRISPR–Cas9

(2015) Ophir Shalem et al.   NATURE REVIEWS GENETICS

Beyond editing: repurposing CRISPR–Cas9 for precision genome regulation and interrogation

(2015) Antonia A. Dominguez et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling

(2014) Randall J. Platt et al.   CELL

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

(2014) S. Kim et al.   GENOME RESEARCH

In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Inhibition of polo-like kinase 1 in glioblastoma multiforme induces mitotic catastrophe and enhances radiosensitisation

(2013) Anita T. Tandle et al.   EUROPEAN JOURNAL OF CANCER

Macromolecule Functionalization of Disulfide-Bonded Polymer Hydrogel Capsules and Cancer Cell Targeting

(2012) Olga Shimoni et al.   ACS Nano

Targeted Delivery of PLK1-siRNA by ScFv Suppresses Her2+ Breast Cancer Growth and Metastasis

(2012) Y.-d. Yao et al.   Science Translational Medicine

Cellular Association and Cargo Release of Redox-Responsive Polymer Capsules Mediated by Exofacial Thiols

(2011) Yan Yan et al.   ADVANCED MATERIALS

Biodegradable poly(amine-co-ester) terpolymers for targeted gene delivery

(2011) Jiangbing Zhou et al.   NATURE MATERIALS

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

(2011) H. Cabral et al.   Nature Nanotechnology