Evidence map›Paper›PMID 30623253›Full record

ArticlePharmaceutical research2019

Application of a Quality-By-Design Approach to Optimise Lipid-Polymer Hybrid Nanoparticles Loaded with a Splice-Correction Antisense Oligonucleotide: Maximising Loading and Intracellular Delivery.

Kaushik Thanki, Simon Papai, Abhijeet Lokras, Fabrice Rose, Emily Falkenberg, Henrik Franzyk, Camilla Foged

Abstract read
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In one paragraph

Article in Pharmaceutical research, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed, 1 pooled it
0.9field-weighted citation impact, top 28% 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

8 citing papers in PubMed, 1 synthesis or guideline pooled it, 17 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Review
  4. Review
  5. Review
  6. Article
  7. Article
  8. 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

7 authors at 1 institution in 1 country.

Kaushik ThankiDepartment of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark.
Simon PapaiDepartment of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark.
Abhijeet LokrasDepartment of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark.
Fabrice RoseDepartment of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark.
Emily FalkenbergDepartment of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark.
Henrik FranzykDepartment of Drug Design and Pharmacology, Faculty of Health and Medical Sciences,, University of Copenhagen, Jagtvej 162, DK-2100, Copenhagen Ø, Denmark.
Camilla FogedDepartment of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Universitetsparken 2,, DK-2100, Copenhagen Ø, Denmark. camilla.foged@sund.ku.dk.ORCID http://orcid.org/0000-0003-2812-5588
University of Copenhagen · DK

Funding

Innovative Medicines Initiative 115363Lundbeckfonden (DK) R219-2016-908Novo Nordisk Fonden NNF17OC0026526Seventh Framework Programme 600207
6 · The paper itself

Abstract

backgroundAntisense oligonucleotides (ASOs) are promising therapeutics for specific modulation of cellular RNA function. However, ASO efficacy is compromised by inefficient intracellular delivery. Lipid-polymer hybrid nanoparticles (LPNs) are attractive mediators of intracellular ASO delivery due to favorable colloidal stability and sustained release properties.

methodsLPNs composed of cationic lipidoid 5 (L

resultsA series of CQAs were identified based on the quality target product profile. The L

conclusionsThe optimal Luc-ASO-loaded LPNs represent a robust formulation that mediates efficient intracellular delivery of Luc-ASO. This opens new avenues for further development of LPNs as a broadly applicable technology platform for delivering nucleic acid cargos intracellularly.

Indexed as

Drug Delivery SystemsDelayed-Action PreparationsGenetic TherapyHeLa CellsHumansLipidsLuciferasesNanoparticlesOligonucleotides, AntisensePolylactic Acid-Polyglycolic Acid CopolymerRNA SplicingTechnology, PharmaceuticalDelayed-Action PreparationsLipidsLuciferasesOligonucleotides, AntisensePolylactic Acid-Polyglycolic Acid Copolymerantisense oligonucleotidesHeLa pLuc/705 cellsin vitro splice correctionlipidoidslipid-polymer hybrid nanoparticles (LPNs)quality-by-designstatistical optimisation

Identifiers

PMID30623253
OpenAlexW2908810665

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.