Evidence map›Paper›PMID 32937374›Full record

ArticleScience advances2020

Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing.

Xinfu Zhang, Weiyu Zhao, Giang N Nguyen, Chengxiang Zhang, Chunxi Zeng, Jingyue Yan, Shi Du, Xucheng Hou, Wenqing Li, Justin Jiang and 10 more

Open access · goldAbstract read
In one paragraph

Article in Science advances, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 101 papers.

0numbers the graph read from it
0cells of the map it votes in
101citing papers in PubMed
8.3field-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

101 citing papers in PubMed, 175 citations in OpenAlex.

  1. Article
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  6. Review
  7. The Role of CRISPR and Its Therapeutic Applications in Glioblastoma.International journal of molecular sciences · 2026
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  11. Article
  12. Review
  13. Research progress of mRNA vaccines for infectious diseases.European journal of medical research · 2025
    Review
  14. Review
  15. Article
  16. Unlock the sustained therapeutic efficacy of mRNA.Journal of controlled release : official journal of the Controlled Release Society · 2025
    Review
  17. Article
  18. Article
  19. Article
  20. Article

41 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

20 authors at 5 institutions in 2 countries.

Xinfu ZhangDivision of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, OH 43210, USA.ORCID 0000-0001-5544-1522
Weiyu ZhaoDivision of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, OH 43210, USA.ORCID 0000-0003-3345-4925
Giang N NguyenThe Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.ORCID 0000-0003-3205-9106
Chengxiang ZhangDivision of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, OH 43210, USA.
Chunxi ZengDivision of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, OH 43210, USA.ORCID 0000-0002-1218-1908
Jingyue YanDivision of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, OH 43210, USA.
Shi DuDivision of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, OH 43210, USA.
Xucheng HouDivision of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, OH 43210, USA.
Wenqing LiDivision of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, OH 43210, USA.
Justin JiangDivision of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, OH 43210, USA.
Binbin DengCenter for Electron Microscopy and Analysis, Department of Materials Science and Engineering, The Ohio State University, Columbus, OH 43210, USA.
David W McCombCenter for Electron Microscopy and Analysis, Department of Materials Science and Engineering, The Ohio State University, Columbus, OH 43210, USA.ORCID 0000-0003-3763-9652
Robert DorkinBeam Therapeutics, Cambridge, MA 02139, USA.
Aalok ShahBeam Therapeutics, Cambridge, MA 02139, USA.ORCID 0000-0001-5601-088X
Luis BarreraBeam Therapeutics, Cambridge, MA 02139, USA.ORCID 0000-0003-0612-6416
Francine GregoireBeam Therapeutics, Cambridge, MA 02139, USA.
Manmohan SinghBeam Therapeutics, Cambridge, MA 02139, USA.
Delai ChenBeam Therapeutics, Cambridge, MA 02139, USA. dong.525@osu.edu dsabatin@pennmedicine.upenn.edu dchen@beamtx.com.ORCID 0000-0002-7936-7889
Denise E SabatinoThe Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. dong.525@osu.edu dsabatin@pennmedicine.upenn.edu dchen@beamtx.com.ORCID 0000-0002-6116-5453
Yizhou DongDivision of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, OH 43210, USA. dong.525@osu.edu dsabatin@pennmedicine.upenn.edu dchen@beamtx.com.ORCID 0000-0001-5786-0659
The Ohio State University · USBeam Therapeutics (United States) · USChildren's Hospital of Philadelphia · USDalian University of Technology · CNLung Institute · US

Funding

Biodegradable nanoparticles, a genome editing platform to treat hemophiliaR01HL136652 · NHLBI · OHIO STATE UNIVERSITY · PI DONG, YIZHOU · 2017 to 2021
$2.3M
Cell-specific and multifunctional drug formulations for in vivo deliveryR35GM119679 · NIGMS · OHIO STATE UNIVERSITY · PI DONG, YIZHOU · 2016 to 2020
$1.9M
NHLBI NIH HHS R01 HL136652NIGMS NIH HHS R35 GM119679
6 · The paper itself

Abstract

Messenger RNA (mRNA) therapeutics have been explored to treat various genetic disorders. Lipid-derived nanomaterials are currently one of the most promising biomaterials that mediate effective mRNA delivery. However, efficiency and safety of this nanomaterial-based mRNA delivery remains a challenge for clinical applications. Here, we constructed a series of lipid-like nanomaterials (LLNs), named functionalized TT derivatives (FTT), for mRNA-based therapeutic applications in vivo. After screenings on the materials, we identified FTT5 as a lead material for efficient delivery of long mRNAs, such as human factor VIII (hFVIII) mRNA (~4.5 kb) for expression of hFVIII protein in hemophilia A mice. Moreover, FTT5 LLNs demonstrated high percentage of base editing on PCSK9 in vivo at a low dose of base editor mRNA (~5.5 kb) and single guide RNA. Consequently, FTT nanomaterials merit further development for mRNA-based therapy.

Indexed as

NanoparticlesProprotein Convertase 9AnimalsGene EditingLipidsMiceRNA, MessengerLipidsPCSK9 protein, humanProprotein Convertase 9RNA, Messenger

Identifiers

PMID32937374
PMCPMC7442477
OpenAlexW3081421945

What OpenQuestion holds

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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.