Evidence map›Paper›PMID 31140918›Full record

ArticleNucleic acid therapeutics2019

Formulation of Biocompatible Targeted ECO/siRNA Nanoparticles with Long-Term Stability for Clinical Translation of RNAi.

Nadia R Ayat, Zhanhu Sun, Da Sun, Michelle Yin, Ryan C Hall, Amita M Vaidya, Xujie Liu, Andrew L Schilb, Josef H Scheidt, Zheng-Rong Lu

Abstract read
In one paragraph

Article in Nucleic acid therapeutics, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.

0numbers the graph read from it
0cells of the map it votes in
21citing papers in PubMed
–field-weighted citation impact
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

21 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
  4. Review
  5. Article
  6. Article
  7. Article
  8. Review
  9. Review
  10. Review
  11. Article
  12. Review
  13. Effective gene therapy of Stargardt disease with PEG-ECO/Molecular therapy. Nucleic acids · 2022
    Article
  14. Article
  15. Review
  16. Article
  17. Article
  18. mRNA-lipid nanoparticle COVID-19 vaccines: Structure and stability.International journal of pharmaceutics · 2021
    Review
  19. Formulation and efficacy of ECO/pRHO-ABCA4-SV40 nanoparticles for nonviral gene therapy of Stargardt disease in a mouse model.Journal of controlled release : official journal of the Controlled Release Society · 2021
    Article
  20. Article
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

10 authors.

Nadia R AyatDepartment of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, Ohio.
Zhanhu SunDepartment of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, Ohio.
Da SunDepartment of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, Ohio.
Michelle YinDepartment of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, Ohio.
Ryan C HallDepartment of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, Ohio.
Amita M VaidyaDepartment of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, Ohio.
Xujie LiuDepartment of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, Ohio.
Andrew L SchilbDepartment of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, Ohio.
Josef H ScheidtDepartment of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, Ohio.
Zheng-Rong LuDepartment of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, Ohio.

Funding

Smart nanoparticles regulating oncogenic IncRNA for breast cancer therapyR01CA235152 · NCI · CASE WESTERN RESERVE UNIVERSITY · PI ZHENG-RONG LU, William Schiemann · 2019 to 2026
$3.2M
Image-guided EMT inhibition for treating metastatic breast cancerR01CA194518 · NCI · CASE WESTERN RESERVE UNIVERSITY · PI LU, ZHENG-RONG, SCHIEMANN, WILLIAM · 2015 to 2019
$2.4M
CCR NIH HHS HHSN261200800001CNCI NIH HHS HHSN261200800001ENCI NIH HHS R01 CA194518NCI NIH HHS R01 CA235152
6 · The paper itself

Abstract

Nanoparticle based siRNA formulations often suffer from aggregation and loss of function during storage. We in this study report a frozen targeted RGD-polyethylene glycol (PEG)-ECO/siβ3 nanoparticle formulation with a prolonged shelf life and preserved nanoparticle functionality. The targeted RGD-PEG-ECO/siβ3 nanoparticles are formed by step-wised self-assembly of RGD-PEG-maleimide, ECO, and siRNA. The nanoparticles have a diameter of 224.5 ± 9.41 nm and a zeta potential to 45.96 ± 3.67 mV in water and a size of 234.34 ± 3.01 nm and a near neutral zeta potential in saline solution. The addition of sucrose does not affect their size and zeta potential and substantially preserves the integrity and biological activities of frozen and lyophilized formulations of the targeted nanoparticles. The frozen formulation with as low as 5% sucrose retains nanoparticle integrity (90% siRNA encapsulation), size distribution (polydispersity index [PDI] ≤20%), and functionality (at least 75% silencing efficiency) at -80°C for at least 1 year. The frozen RGD-PEG-ECO/siβ3 nanoparticle formulation exhibits excellent biocompatibility, with no adverse effects on hemocompatibility and minimal immunogenicity. As RNAi holds the promise in treating the previously untreatable diseases, the frozen nanoparticle formulation with the low sucrose concentration has the potential to be a delivery platform for clinical translation of RNAi therapeutics.

Indexed as

Drug Delivery SystemsBiocompatible MaterialsCell Line, TumorHumansNanoparticlesPolyethylene GlycolsRNA, Double-StrandedRNA InterferenceRNA, Small InterferingBiocompatible MaterialsPolyethylene GlycolsRNA, Double-StrandedRNA, Small InterferingbiocompatibilityECOlong-term stabilityRNAisiRNA nanoparticles

Identifiers

PMID31140918
PMCPMC6686697

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.