mRNA‐Loaded Lipid‐Like Nanoparticles for Liver Base Editing Via the Optimization of Central Composite Design

Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

(2020) Jonathan M. Levy et al.   Nature Biomedical Engineering

Protein and mRNA Delivery Enabled by Cholesteryl‐Based Biodegradable Lipidoid Nanoparticles

(2020) Yamin Li et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

(2020) Qiang Cheng et al.   Nature Nanotechnology

Chemical modifications of adenine base editor mRNA and guide RNA expand its application scope

(2020) Tingting Jiang et al.   Nature Communications

Imidazole‐based Synthetic Lipidoids for In Vivo mRNA Delivery into Primary T Lymphocytes

(2020) Xuewei Zhao et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

A Potent Branched-Tail Lipid Nanoparticle Enables Multiplexed mRNA Delivery and Gene Editing In Vivo

(2020) Khalid A. Hajj et al.   NANO LETTERS

Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing

(2020) Tuo Wei et al.   Nature Communications

Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing

(2020) Xinfu Zhang et al.   Science Advances

Metabolic rewiring of macrophages by CpG potentiates clearance of cancer cells and overcomes tumor-expressed CD47−mediated ‘don’t-eat-me’ signal

(2019) Mingen Liu et al.   NATURE IMMUNOLOGY

Adenine base editing in an adult mouse model of tyrosinaemia

(2019) Chun-Qing Song et al.   Nature Biomedical Engineering

Delivery Aspects of CRISPR/Cas for in Vivo Genome Editing

(2019) Danny Wilbie et al.   ACCOUNTS OF CHEMICAL RESEARCH

Understanding Cell Penetration of Cyclic Peptides

(2019) Patrick G. Dougherty et al.   CHEMICAL REVIEWS

The next generation of CRISPR–Cas technologies and applications

(2019) Adrian Pickar-Oliver et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

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

(2019) Ji Liu et al.   ADVANCED MATERIALS

Delivery of mRNA vaccines with heterocyclic lipids increases anti-tumor efficacy by STING-mediated immune cell activation

(2019) Lei Miao et al.   NATURE BIOTECHNOLOGY

RNA delivery biomaterials for the treatment of genetic and rare diseases

(2019) Weiyu Zhao et al.   BIOMATERIALS

Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening

(2019) Pedro P.G. Guimaraes et al.   JOURNAL OF CONTROLLED RELEASE

Co-delivery of mRNA and SPIONs through amino-ester nanomaterials

(2018) Xiao Luo et al.   Nano Research

Rapid, Single-Cell Analysis and Discovery of Vectored mRNA Transfection In Vivo with a loxP-Flanked tdTomato Reporter Mouse

(2018) Kevin J. Kauffman et al.   Molecular Therapy-Nucleic Acids

Optimization of a Degradable Polymer–Lipid Nanoparticle for Potent Systemic Delivery of mRNA to the Lung Endothelium and Immune Cells

(2018) James C. Kaczmarek et al.   NANO LETTERS

Optimization of the Linker Length of Mannose-Cholesterol Conjugates for Enhanced mRNA Delivery to Dendritic Cells by Liposomes

(2018) Fazhan Wang et al.   Frontiers in Pharmacology

Dendrimer-Based Lipid Nanoparticles Deliver Therapeutic FAH mRNA to Normalize Liver Function and Extend Survival in a Mouse Model of Hepatorenal Tyrosinemia Type I

(2018) Qiang Cheng et al.   ADVANCED MATERIALS

Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

(2018) Lukas Villiger et al.   NATURE MEDICINE

In situ sprayed bioresponsive immunotherapeutic gel for post-surgical cancer treatment

(2018) Qian Chen et al.   Nature Nanotechnology

Base editing: precision chemistry on the genome and transcriptome of living cells

(2018) Holly A. Rees et al.   NATURE REVIEWS GENETICS

High-throughput in vivo screen of functional mRNA delivery identifies nanoparticles for endothelial cell gene editing

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

Glycyrrhetinic Acid Liposomes Containing Mannose-Diester Lauric Diacid-Cholesterol Conjugate Synthesized by Lipase-Catalytic Acylation for Liver-Specific Delivery

(2017) Jing Chen et al.   MOLECULES

Delivery technologies for genome editing

(2017) Hao Yin et al.   NATURE REVIEWS DRUG DISCOVERY

Tools for translation: non-viral materials for therapeutic mRNA delivery

(2017) Khalid A. Hajj et al.   Nature Reviews Materials

A Single Methylene Group in Oligoalkylamine-Based Cationic Polymers and Lipids Promotes Enhanced mRNA Delivery

(2016) Anita Jarzębińska et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

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

Lipid Nanoparticle Assisted mRNA Delivery for Potent Cancer Immunotherapy

(2016) Matthias A. Oberli et al.   NANO LETTERS

Systemic RNA delivery to dendritic cells exploits antiviral defence for cancer immunotherapy

(2016) Lena M. Kranz et al.   NATURE

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo

(2015) Bin Li et al.   NANO LETTERS

Optimization of Lipid Nanoparticle Formulations for mRNA Delivery in Vivo with Fractional Factorial and Definitive Screening Designs

(2015) Kevin J. Kauffman et al.   NANO LETTERS

Experimental design methods for bioengineering applications

(2014) Tuğba Keskin Gündoğdu et al.   CRITICAL REVIEWS IN BIOTECHNOLOGY

mRNA-based therapeutics — developing a new class of drugs

(2014) Ugur Sahin et al.   NATURE REVIEWS DRUG DISCOVERY

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Enhancing tumor cell response to chemotherapy through nanoparticle-mediated codelivery of siRNA and cisplatin prodrug

(2013) X. Xu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Minimal "Self" Peptides That Inhibit Phagocytic Clearance and Enhance Delivery of Nanoparticles

(2013) P. L. Rodriguez et al.   SCIENCE

Helical poly(arginine) mimics with superior cell-penetrating and molecular transporting properties

(2013) Haoyu Tang et al.   Chemical Science

Cell-Penetrating Homochiral Cyclic Peptides as Nuclear-Targeting Molecular Transporters

(2011) Deendayal Mandal et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Phenylketonuria

(2010) Nenad Blau et al.   LANCET

Inhibition of “self” engulfment through deactivation of myosin-II at the phagocytic synapse between human cells

(2008) Richard K. Tsai et al.   JOURNAL OF CELL BIOLOGY