Evidence map›Paper›PMID 28202722›Full record

ArticleProceedings of the National Academy of Sciences of the United States of America2017

Systemic delivery of factor IX messenger RNA for protein replacement therapy.

Suvasini Ramaswamy, Nina Tonnu, Kiyoshi Tachikawa, Pattraranee Limphong, Jerel B Vega, Priya P Karmali, Pad Chivukula, Inder M Verma

Open access · hybridAbstract read
In one paragraph

Article in Proceedings of the National Academy of Sciences of the United States of America, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 165 papers.

0numbers the graph read from it
0cells of the map it votes in
165citing papers in PubMed
11.1field-weighted citation impact, top 1% 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

165 citing papers in PubMed, 296 citations in OpenAlex.

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  15. Research progress of mRNA vaccines for infectious diseases.European journal of medical research · 2025
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105 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

8 authors at 2 institutions in 1 country.

Suvasini RamaswamyLaboratory of Genetics, Salk Institute for Biological Studies, La Jolla, CA 92037.
Nina TonnuLaboratory of Genetics, Salk Institute for Biological Studies, La Jolla, CA 92037.
Kiyoshi TachikawaArcturus Therapeutics, San Diego, CA 92121.
Pattraranee LimphongArcturus Therapeutics, San Diego, CA 92121.
Jerel B VegaArcturus Therapeutics, San Diego, CA 92121.
Priya P KarmaliArcturus Therapeutics, San Diego, CA 92121.
Pad ChivukulaArcturus Therapeutics, San Diego, CA 92121.
Inder M VermaLaboratory of Genetics, Salk Institute for Biological Studies, La Jolla, CA 92037; verma@salk.edu.
Arcturus Therapeutics (United States) · USSalk Institute for Biological Studies · US

Funding

Viral Vector Core (VVC)P30CA014195 · NCI · SALK INSTITUTE FOR BIOLOGICAL STUDIES · PI Ye Zheng · 1985 to 2026
$82.8M
Molecular Mechanisms and Therapeutic Approaches to Malignant GliomasR01CA195613 · NCI · SALK INSTITUTE FOR BIOLOGICAL STUDIES · PI HUNTER, TONY R. · 2016 to 2019
$2.7M
NCI NIH HHS P30 CA014195NCI NIH HHS R01 CA195613
6 · The paper itself

Abstract

Safe and efficient delivery of messenger RNAs for protein replacement therapies offers great promise but remains challenging. In this report, we demonstrate systemic, in vivo, nonviral mRNA delivery through lipid nanoparticles (LNPs) to treat a Factor IX (

Indexed as

AnimalsCholesterolCytokinesDisease Models, AnimalDrug CarriersDrug CompoundingDrug Evaluation, PreclinicalFactor IXFemaleGenetic TherapyHemophilia BHumansHydrogen-Ion ConcentrationInjections, IntravenousLiverMale1,2-distearoyllecithinCholesterolCytokinesDrug CarriersFactor IXPhosphatidylcholinesRecombinant ProteinsRNA, Messengerhemophilia B therapyhepatic diseaseslipid nanoparticlesnonviral mRNA deliverysystemic delivery

Identifiers

PMID28202722
PMCPMC5347596
OpenAlexW2587871737

What OpenQuestion holds

Textmetadata
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.