Augmented lipid-nanoparticle-mediated in vivo genome editing in the lungs and spleen by disrupting Cas9 activity in the liver

Drug delivery systems for RNA therapeutics

(2022) Kalina Paunovska et al.   NATURE REVIEWS GENETICS

Membrane-destabilizing ionizable phospholipids for organ-selective mRNA delivery and CRISPR–Cas gene editing

(2021) Shuai Liu et al.   NATURE MATERIALS

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

(2021) Kiran Musunuru et al.   NATURE

In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels

(2021) Tanja Rothgangl et al.   NATURE BIOTECHNOLOGY

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2020) Qiang Cheng et al.   Nature Nanotechnology

LB002ILLUMINATE-A, A PHASE 3 STUDY OF LUMASIRAN, AN INVESTIGATIONAL RNAI THERAPEUTIC, IN CHILDREN AND ADULTS WITH PRIMARY HYPEROXALURIA TYPE 1 (PH1)

(2020) Sander Garrelfs et al.   NEPHROLOGY DIALYSIS TRANSPLANTATION

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

(2020) Manisha Balwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2020) Xinfu Zhang et al.   Science Advances

Treating Disease at the RNA Level with Oligonucleotides

(2019) Arthur A. Levin   NEW ENGLAND JOURNAL OF MEDICINE

A New Class of Medicines through DNA Editing

(2019) Matthew H. Porteus   NEW ENGLAND JOURNAL OF MEDICINE

A High-Throughput Platform to Identify Small-Molecule Inhibitors of CRISPR-Cas9

(2019) Basudeb Maji et al.   CELL

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

(2019) Julia F. Alterman et al.   NATURE BIOTECHNOLOGY

Tissue-restricted Genome Editing in vivo Specified by MicroRNA-repressible Anti-CRISPR Proteins

(2019) Jooyoung Lee et al.   RNA

Ligand Conjugated Multimeric siRNAs Enable Enhanced Uptake and Multiplexed Gene Silencing

(2019) Jonathan M. Brown et al.   Nucleic Acid Therapeutics

Functional Insights Revealed by the Kinetic Mechanism of CRISPR/Cas9

(2018) Austin T. Raper et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Reversal of siRNA-mediated gene silencing in vivo

(2018) Ivan Zlatev et al.   NATURE BIOTECHNOLOGY

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

(2018) Jonathan D. Finn et al.   Cell Reports

Endothelial siRNA delivery in nonhuman primates using ionizable low–molecular weight polymeric nanoparticles

(2018) Omar F. Khan et al.   Science Advances

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

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

Synthetic Oligonucleotides Inhibit CRISPR-Cpf1-Mediated Genome Editing

(2018) Bin Li et al.   Cell Reports

Efficient Liver Targeting by Polyvalent Display of a Compact Ligand for the Asialoglycoprotein Receptor

(2017) Carlos A. Sanhueza et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Lipid Nanoparticle Systems for Enabling Gene Therapies

(2017) Pieter R. Cullis et al.   MOLECULAR THERAPY

Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

(2017) Xue Gao et al.   NATURE

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

(2017) Hao Yin et al.   NATURE BIOTECHNOLOGY

Cpf1 proteins excise CRISPR RNAs from mRNA transcripts in mammalian cells

(2017) Guocai Zhong et al.   Nature Chemical Biology

Inclisiran in Patients at High Cardiovascular Risk with Elevated LDL Cholesterol

(2017) Kausik K. Ray et al.   NEW ENGLAND JOURNAL OF MEDICINE

Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs

(2017) Xiulong Shen et al.   NUCLEIC ACIDS RESEARCH

Disabling Cas9 by an anti-CRISPR DNA mimic

(2017) Jiyung Shin et al.   Science Advances

Engineering CRISPR–Cpf1 crRNAs and mRNAs to maximize genome editing efficiency

(2017) Bin Li et al.   Nature Biomedical Engineering

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

(2016) Jason B. Miller et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Naturally Occurring Off-Switches for CRISPR-Cas9

(2016) April Pawluk et al.   CELL

Mechanism of hard-nanomaterial clearance by the liver

(2016) Kim M. Tsoi et al.   NATURE MATERIALS

Improving the efficacy and safety of biologic drugs with tolerogenic nanoparticles

(2016) Takashi K. Kishimoto et al.   Nature Nanotechnology

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

(2015) Bernd Zetsche et al.   CELL

Going beyond the liver: Progress and challenges of targeted delivery of siRNA therapeutics

(2015) Cornelia Lorenzer et al.   JOURNAL OF CONTROLLED RELEASE

Principles of nanoparticle design for overcoming biological barriers to drug delivery

(2015) Elvin Blanco et al.   NATURE BIOTECHNOLOGY

An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia

(2015) Alfica Sehgal et al.   NATURE MEDICINE

Toehold Switches: De-Novo-Designed Regulators of Gene Expression

(2014) Alexander A. Green et al.   CELL

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling

(2014) Randall J. Platt et al.   CELL

Interplay of LNA and 2′-O-Methyl RNA in the Structure and Thermodynamics of RNA Hybrid Systems: A Molecular Dynamics Study Using the Revised AMBER Force Field and Comparison with Experimental Results

(2014) Ilyas Yildirim et al.   JOURNAL OF PHYSICAL CHEMISTRY B

In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight

(2014) James E. Dahlman et al.   Nature Nanotechnology

Easy quantitative assessment of genome editing by sequence trace decomposition

(2014) Eva K. Brinkman et al.   NUCLEIC ACIDS RESEARCH

Small RNA combination therapy for lung cancer

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

Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates

(2014) Y. Dong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Safety profile of RNAi nanomedicines

(2012) Scott A. Barros et al.   ADVANCED DRUG DELIVERY REVIEWS

Designing Chemically Modified Oligonucleotides for Targeted Gene Silencing

(2012) Glen F. Deleavey et al.   CHEMISTRY & BIOLOGY

Rapid Discovery of Potent siRNA-Containing Lipid Nanoparticles Enabled by Controlled Microfluidic Formulation

(2012) Delai Chen et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

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

(2012) M. Jinek et al.   SCIENCE

Systemic RNAi-mediated Gene Silencing in Nonhuman Primate and Rodent Myeloid Cells

(2012) Tatiana I Novobrantseva et al.   Molecular Therapy-Nucleic Acids

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Lipid-like materials for low-dose, in vivo gene silencing

(2010) K. T. Love et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Stability Study of Unmodified siRNA and Relevance to Clinical Use

(2008) Robyn P. Hickerson et al.   OLIGONUCLEOTIDES