Piperazine-derived lipid nanoparticles deliver mRNA to immune cells in vivo

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

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

Drug delivery systems for RNA therapeutics

(2022) Kalina Paunovska et al.   NATURE REVIEWS GENETICS

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

(2022) Cory D. Sago et al.   Nature Biomedical Engineering

Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

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

Conformation-sensitive targeting of lipid nanoparticles for RNA therapeutics

(2021) Niels Dammes et al.   Nature Nanotechnology

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

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

Non-liver mRNA Delivery

(2021) David Loughrey et al.   ACCOUNTS OF CHEMICAL RESEARCH

A Combinatorial Library of Lipid Nanoparticles for RNA Delivery to Leukocytes

(2020) Srinivas Ramishetti et al.   ADVANCED MATERIALS

Ionizable Lipid Nanoparticle-Mediated mRNA Delivery for Human CAR T Cell Engineering

(2020) Margaret M. Billingsley et al.   NANO LETTERS

Optimizing synthetic nucleic acid and protein nanocarriers: The chemical evolution approach

(2020) Franziska Freitag et al.   ADVANCED DRUG DELIVERY REVIEWS

A Nanoprimer To Improve the Systemic Delivery of siRNA and mRNA

(2020) Nell R. M. Saunders et al.   NANO LETTERS

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

Nanoparticles containing constrained phospholipids deliver mRNA to liver immune cells in vivo without targeting ligands

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An appraisal of anti-mycobacterial activity with structure-activity relationship of piperazine and its analogues: A review

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Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine

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

Nanoparticles Containing Oxidized Cholesterol Deliver mRNA to the Liver Microenvironment at Clinically Relevant Doses

(2019) Kalina Paunovska et al.   ADVANCED MATERIALS

Mild Innate Immune Activation Overrides Efficient Nanoparticle‐Mediated RNA Delivery

(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

A modular platform for targeted RNAi therapeutics

(2018) Ranit Kedmi et al.   Nature Nanotechnology

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

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

Modifying a Commonly Expressed Endocytic Receptor Retargets Nanoparticles in Vivo

(2018) Cory D. Sago et al.   NANO LETTERS

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

Correction for Sago et al., High-throughput in vivo screen of functional mRNA delivery identifies nanoparticles for endothelial cell gene editing

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Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes

(2018) Nuphar Veiga et al.   Nature Communications

Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery

(2016) Owen S. Fenton et al.   ADVANCED MATERIALS

Mechanism of hard-nanomaterial clearance by the liver

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

Piperazine scaffold: A remarkable tool in generation of diverse pharmacological agents

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

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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

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

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

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

Correlating animal and human phase Ia/Ib clinical data with CALAA-01, a targeted, polymer-based nanoparticle containing siRNA

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Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo

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

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Combinatorial synthesis of chemically diverse core-shell nanoparticles for intracellular delivery

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Ciprofloxacin: review on developments in synthetic, analytical, and medicinal aspects

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

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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

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Development of Lipidoid–siRNA Formulations for Systemic Delivery to the Liver

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A combinatorial library of lipid-like materials for delivery of RNAi therapeutics

(2008) Akin Akinc et al.   NATURE BIOTECHNOLOGY