Universal Barcoding Predicts In Vivo ApoE-Independent Lipid Nanoparticle Delivery

Species-dependent in vivo mRNA delivery and cellular responses to nanoparticles

(2022) Marine Z. C. Hatit et al.   Nature Nanotechnology

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

Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

(2021) Sander F. Garrelfs et al.   NEW ENGLAND JOURNAL OF MEDICINE

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

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

On the mechanism of tissue-specific mRNA delivery by selective organ targeting nanoparticles

(2021) Sean A. Dilliard et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2020) Qiang Cheng et al.   Nature Nanotechnology

Two Phase 3 Trials of Inclisiran in Patients with Elevated LDL Cholesterol

(2020) Kausik K. Ray et al.   NEW ENGLAND JOURNAL OF MEDICINE

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

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

Efficacy and Safety of the mRNA-1273 SARS-CoV-2 Vaccine

(2020) Lindsey R. Baden et al.   NEW ENGLAND JOURNAL OF MEDICINE

Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine

(2020) Fernando P. Polack et al.   NEW ENGLAND JOURNAL OF MEDICINE

Using Large Datasets to Understand Nanotechnology

(2019) Kalina Paunovska et al.   ADVANCED MATERIALS

Constrained Nanoparticles Deliver siRNA and sgRNA to T Cells In Vivo without Targeting Ligands

(2019) Melissa P. Lokugamage et al.   ADVANCED MATERIALS

The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

(2019) Akin Akinc et al.   Nature Nanotechnology

Analyzing 2000 in Vivo Drug Delivery Data Points Reveals Cholesterol Structure Impacts Nanoparticle Delivery

(2018) Kalina Paunovska et al.   ACS Nano

A Direct Comparison of in Vitro and in Vivo Nucleic Acid Delivery Mediated by Hundreds of Nanoparticles Reveals a Weak Correlation

(2018) Kalina Paunovska et al.   NANO LETTERS

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

Nanoparticles That Deliver RNA to Bone Marrow Identified by in Vivo Directed Evolution

(2018) Cory D. Sago et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

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

Tissue-based map of the human proteome

(2015) M. Uhlen et al.   SCIENCE

Tartrate-resistant acid phosphatase (TRAP) co-localizes with receptor activator of NF-KB ligand (RANKL) and osteoprotegerin (OPG) in lysosomal-associated membrane protein 1 (LAMP1)-positive vesicles in rat osteoblasts and osteocytes

(2014) L. B. Solberg et al.   HISTOCHEMISTRY AND CELL BIOLOGY

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

(2014) Jayaprakash K. Nair et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

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

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

Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape

(2013) Jerome Gilleron et al.   NATURE BIOTECHNOLOGY

Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles

(2013) Barbara L Mui et al.   Molecular Therapy-Nucleic Acids

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

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

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

(2010) Akin Akinc et al.   MOLECULAR THERAPY