Evidence map›Paper›PMID 32496069›Full record

ArticleNano letters2020

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

Khalid A Hajj, Jilian R Melamed, Namit Chaudhary, Nicholas G Lamson, Rebecca L Ball, Saigopalakrishna S Yerneni, Kathryn A Whitehead

Open access · greenAbstract read
In one paragraph

Article in Nano letters, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 84 papers.

0numbers the graph read from it
0cells of the map it votes in
84citing papers in PubMed
5.7field-weighted citation impact, top 3% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

84 citing papers in PubMed, 138 citations in OpenAlex.

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  18. Harnessing mRNA-lipid nanoparticles as innovative therapies for autoimmune diseases.Molecular therapy. Methods & clinical development · 2025
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24 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors at 1 institution in 1 country.

Khalid A HajjDepartment of Chemical Engineering, Carnegie Mellon University, Pittsburgh, Pennsylvania 15213, United States.
Jilian R MelamedDepartment of Chemical Engineering, Carnegie Mellon University, Pittsburgh, Pennsylvania 15213, United States.
Namit ChaudharyDepartment of Chemical Engineering, Carnegie Mellon University, Pittsburgh, Pennsylvania 15213, United States.
Nicholas G LamsonDepartment of Chemical Engineering, Carnegie Mellon University, Pittsburgh, Pennsylvania 15213, United States.
Rebecca L BallDepartment of Chemical Engineering, Carnegie Mellon University, Pittsburgh, Pennsylvania 15213, United States.
Saigopalakrishna S YerneniDepartment of Biomedical Engineering, Carnegie Mellon University, Pittsburgh, Pennsylvania 15213, United States.
Kathryn A WhiteheadDepartment of Chemical Engineering, Carnegie Mellon University, Pittsburgh, Pennsylvania 15213, United States.ORCID 0000-0002-0100-7824
Carnegie Mellon University · US

Funding

Engineering Smart Protein Coronas to Advance Theraputic mRNA DeliveryF32EB029345 · NIBIB · CARNEGIE-MELLON UNIVERSITY · PI MELAMED, JILIAN R · 2019 to 2021
$128k
NIBIB NIH HHS F32 EB029345
6 · The paper itself

Abstract

The clinical translation of messengerRNA (mRNA) drugs has been slowed by a shortage of delivery vehicles that potently and safely shuttle mRNA into target cells. Here, we describe the properties of a particularly potent branched-tail lipid nanoparticle that delivers mRNA to >80% of three major liver cell types. We characterize mRNA delivery spatially, temporally, and as a function of injection type. Following intravenous delivery, our lipid nanoparticle induced greater protein expression than two benchmark lipids, C12-200 and DLin-MC3-DMA, at an mRNA dose of 0.5 mg/kg. Lipid nanoparticles were sufficiently potent to codeliver three distinct mRNAs (firefly luciferase, mCherry, and erythropoietin) and, separately, Cas9 mRNA and single guide RNA (sgRNA) for proof-of-concept nonviral gene editing in mice. Furthermore, our branched-tail lipid nanoparticle was neither immunogenic nor toxic to the liver. Together, these results demonstrate the unique potential of this lipid material to improve the management of diseases rooted in liver dysfunction.

Indexed as

Gene EditingNanoparticlesAnimalsGene Transfer TechniquesLipidsMiceRNA, MessengerLipidsRNA, Messengergene editinglipid nanoparticleslipidoidliver deliverymRNA deliveryprotein expression

Identifiers

PMID32496069
PMCPMC7781386
OpenAlexW3031140223

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.