Evidence map›Paper›PMID 33523869›Full record

ArticleScience advances2021

Ionizable lipid nanoparticles for in utero mRNA delivery.

Rachel S Riley, Meghana V Kashyap, Margaret M Billingsley, Brandon White, Mohamad-Gabriel Alameh, Sourav K Bose, Philip W Zoltick, Hiaying Li, Rui Zhang, Andrew Y Cheng and 3 more

Open access · goldAbstract read
In one paragraph

Article in Science advances, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 116 papers.

0numbers the graph read from it
0cells of the map it votes in
116citing papers in PubMed
12.4field-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

116 citing papers in PubMed, 214 citations in OpenAlex.

  1. Angiogenic Doping: Plausible Yet Difficult to Detect.Sports medicine (Auckland, N.Z.) · 2026
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  20. Therapeutic nanoparticle safety in pregnancy: Bridging knowledge gaps with environmental insights and a translational roadmap.Journal of controlled release : official journal of the Controlled Release Society · 2025
    Review

56 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

13 authors at 3 institutions in 1 country.

Rachel S RileyDepartment of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104, USA.ORCID 0000-0002-3267-5403
Meghana V KashyapThe Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.ORCID 0000-0002-7920-983X
Margaret M BillingsleyDepartment of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104, USA.ORCID 0000-0003-4499-9066
Brandon WhiteThe Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.ORCID 0000-0002-5873-8011
Mohamad-Gabriel AlamehDepartment of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Sourav K BoseThe Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Philip W ZoltickThe Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Hiaying LiThe Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.ORCID 0000-0002-3522-6582
Rui ZhangDepartment of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104, USA.
Andrew Y ChengThe Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.ORCID 0000-0002-1596-8458
Drew WeissmanDepartment of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.ORCID 0000-0002-1501-6510
William H PeranteauThe Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. peranteauw@email.chop.edu mjmitch@seas.upenn.edu.ORCID 0000-0003-1608-861X
Michael J MitchellDepartment of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104, USA. peranteauw@email.chop.edu mjmitch@seas.upenn.edu.ORCID 0000-0002-3628-2244
Children's Hospital of Philadelphia · USUniversity of Pennsylvania · USCalifornia Institute for Regenerative Medicine · US

Funding

Multidisciplinary Training in Cardiovascular BiologyT32HL007954 · NHLBI · UNIVERSITY OF PENNSYLVANIA · PI DAVIES, PETER FRANCIS · 2000 to 2019
$9.9M
In utero gene editing to cure a metabolic liver diseaseR01DK123049 · NIDDK · CHILDREN'S HOSP OF PHILADELPHIA · PI PERANTEAU, WILLIAM H. · 2020 to 2024
$3.7M
In utero CRISPR-mediated gene editing to cure congenital monogenic disordersDP2HL152427 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI PERANTEAU, WILLIAM H. · 2019 to 2019
$2.6M
A data-driven drug delivery (4D) platform for probing and treating the chemoresistant bone marrow microenvironmentDP2TR002776 · NCATS · UNIVERSITY OF PENNSYLVANIA · PI MITCHELL, MICHAEL J · 2018 to 2018
$2.4M
HORMONAL CONTROL OF PROLIFERATION OF MALIGNANT THYMOCYTER01CA024347 · NCI · UNIVERSITY OF TEXAS MEDICAL BR GALVESTON · PI THOMPSON, E AUBREY · 1985 to 2004
$1.8M
Prenatal pulmonary cell gene editing to cure monogenic lung diseasesR01HL151352 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI PERANTEAU, WILLIAM H. · 2020 to 2022
$1.2M
Exosome-mediated enhancement of in utero hematopoietic stem cell transplantation for the treatment of hemoglobinopathiesF32HL143861 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI KASHYAP, MEGHANA · 2018 to 2019
$123k
Advancing mRNA Vaccines for Cancer Therapy Using Molecularly Barcoded NanotechnologyF32CA243475 · NCI · UNIVERSITY OF PENNSYLVANIA · PI RILEY, RACHEL · 2020 to 2020
$9k
NCATS NIH HHS DP2 TR002776NCI NIH HHS F32 CA243475NCI NIH HHS R01 CA024347NHLBI NIH HHS DP2 HL152427NHLBI NIH HHS F32 HL143861NHLBI NIH HHS R01 HL151352NHLBI NIH HHS T32 HL007954NIDDK NIH HHS R01 DK123049
6 · The paper itself

Abstract

Clinical advances enable the prenatal diagnosis of genetic diseases that are candidates for gene and enzyme therapies such as messenger RNA (mRNA)-mediated protein replacement. Prenatal mRNA therapies can treat disease before the onset of irreversible pathology with high therapeutic efficacy and safety due to the small fetal size, immature immune system, and abundance of progenitor cells. However, the development of nonviral platforms for prenatal delivery is nascent. We developed a library of ionizable lipid nanoparticles (LNPs) for in utero mRNA delivery to mouse fetuses. We screened LNPs for luciferase mRNA delivery and identified formulations that accumulate within fetal livers, lungs, and intestines with higher efficiency and safety compared to benchmark delivery systems, DLin-MC3-DMA and jetPEI. We demonstrate that LNPs can deliver mRNAs to induce hepatic production of therapeutic secreted proteins. These LNPs may provide a platform for in utero mRNA delivery for protein replacement and gene editing.

Indexed as

LiposomesNanoparticlesAnimalsGene EditingMiceRNA, MessengerLipid NanoparticlesLiposomesRNA, Messenger

Identifiers

PMID33523869
PMCPMC7806221
OpenAlexW3120748003

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.