Evidence map›Paper›PMID 32622021›Full record

ReviewAdvanced drug delivery reviews2020

Lipid nanoparticle technology for therapeutic gene regulation in the liver.

Dominik Witzigmann, Jayesh A Kulkarni, Jerry Leung, Sam Chen, Pieter R Cullis, Roy van der Meel

Open access · bronzeAbstract readReview
In one paragraph

Review in Advanced drug delivery reviews, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 194 papers.

0numbers the graph read from it
0cells of the map it votes in
194citing papers in PubMed
15.4field-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

194 citing papers in PubMed, 346 citations in OpenAlex.

  1. Cas12a chRDNA-mediatedMolecular therapy. Nucleic acids · 2026
    Article
  2. Review
  3. Review
  4. Review
  5. Dual pKa Lipid Nanoparticles for Lung-tropic mRNA Delivery and pH-Programmed Endosomal Escape.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026
    Article
  6. Article
  7. Review
  8. Review
  9. Review
  10. Review
  11. Review
  12. Article
  13. Article
  14. Article
  15. Review
  16. Article
  17. Review
  18. Reprogramming CD22 CAR-T cells in vivo using CD8-targeted mRNA-LNPs to treat hematological malignancies.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  19. Article
  20. Article

134 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

6 authors at 2 institutions in 2 countries.

Dominik WitzigmannDepartment of Biochemistry and Molecular Biology, University of British Columbia, Vancouver, BC, Canada; NanoMedicines Innovation Network (NMIN), University of British Columbia, Vancouver, BC, Canada.
Jayesh A KulkarniNanoMedicines Innovation Network (NMIN), University of British Columbia, Vancouver, BC, Canada; Centre for Molecular Medicine and Therapeutics, Department of Medical Genetics, BC Children's Hospital Research Institute, University of British Columbia, Vancouver, BC, Canada; Evonik Canada, Vancouver, BC, Canada.
Jerry LeungDepartment of Biochemistry and Molecular Biology, University of British Columbia, Vancouver, BC, Canada.
Sam ChenDepartment of Biochemistry and Molecular Biology, University of British Columbia, Vancouver, BC, Canada; Integrated Nanotherapeutics, Vancouver, BC, Canada.
Pieter R CullisDepartment of Biochemistry and Molecular Biology, University of British Columbia, Vancouver, BC, Canada; NanoMedicines Innovation Network (NMIN), University of British Columbia, Vancouver, BC, Canada. Electronic address: pieterc@mail.ubc.ca.
Roy van der MeelLaboratory of Chemical Biology, Department of Biomedical Engineering and Institute for Complex Molecular Systems, Eindhoven University of Technology, Eindhoven, The Netherlands.
University of British Columbia · CAEindhoven University of Technology · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Hereditary genetic disorders, cancer, and infectious diseases of the liver affect millions of people around the globe and are a major public health burden. Most contemporary treatments offer limited relief as they generally aim to alleviate disease symptoms. Targeting the root cause of diseases originating in the liver by regulating malfunctioning genes with nucleic acid-based drugs holds great promise as a therapeutic approach. However, employing nucleic acid therapeutics in vivo is challenging due to their unfavorable characteristics. Lipid nanoparticle (LNP) delivery technology is a revolutionary development that has enabled clinical translation of gene therapies. LNPs can deliver siRNA, mRNA, DNA, or gene-editing complexes, providing opportunities to treat hepatic diseases by silencing pathogenic genes, expressing therapeutic proteins, or correcting genetic defects. Here we discuss the state-of-the-art LNP technology for hepatic gene therapy including formulation design parameters, production methods, preclinical development and clinical translation.

Indexed as

Gene Expression RegulationGenetic TherapyGene Transfer TechniquesAnimalsHumansLipidsLiverNanoparticlesNanotechnologyLipidsCRISPR/Cas9DNAgene editinggene expressiongene silencingGene therapyguide RNA (gRNA)hepatocytelipid nanoparticle (LNP)lipidslivermessenger RNA (mRNA)small interfering RNA (siRNA)

Identifiers

PMID32622021
PMCPMC7329694
OpenAlexW3040633579

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.