Evidence map›Paper›PMID 37259866›Full record

ReviewArteriosclerosis, thrombosis, and vascular biology2023

New Approaches for Targeting PCSK9: Small-Interfering Ribonucleic Acid and Genome Editing.

Reindert F Oostveen, Amit V Khera, Sekar Kathiresan, Erik S G Stroes, Kevin Fitzgerald, Matthew J Harms, Benjamin L Oakes, John J P Kastelein

Abstract readReview
PubMed Publisher
In one paragraph

Review in Arteriosclerosis, thrombosis, and vascular biology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.

0numbers the graph read from it
0cells of the map it votes in
20citing 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

20 citing papers in PubMed.

  1. Article
  2. mRNA medicine for cardiovascular disease.Nature cardiovascular research · 2026
    Review
  3. Review
  4. Article
  5. Review
  6. Inclisiran as a siRNA Inhibitor of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9); Past, Present, and Future.American journal of cardiovascular drugs : drugs, devices, and other interventions · 2025
    Review
  7. RNA therapeutics in cardiovascular medicine.Current opinion in cardiology · 2025
    Review
  8. Review
  9. Review
  10. Surface-modified extracellular vesicles take their chance in the big PCSK9 inhibitors' race.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  11. Article
  12. Review
  13. Review
  14. EGFR-targeted ionizable lipid nanoparticles enhance in vivo mRNA delivery to the placenta.Journal of controlled release : official journal of the Controlled Release Society · 2024
    Article
  15. Article
  16. Article
  17. Review
  18. Review
  19. Article
  20. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

8 authors.

Reindert F OostveenDepartment of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, The Netherlands (R.F.O., E.S.G.S., J.J.P.K.).ORCID 0000-0001-7182-2886
Amit V KheraVerve Therapeutics, Boston, MA (A.V.K., S.K.).ORCID 0000-0001-6535-5839
Sekar KathiresanVerve Therapeutics, Boston, MA (A.V.K., S.K.).ORCID 0000-0002-3711-7101
Erik S G StroesDepartment of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, The Netherlands (R.F.O., E.S.G.S., J.J.P.K.).ORCID 0000-0001-9555-6260
Kevin FitzgeraldDepartment of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, The Netherlands (R.F.O., E.S.G.S., J.J.P.K.).
Matthew J HarmsScribe Therapeutics, Alameda, CA (M.J.H., B.L.O.).
Benjamin L OakesScribe Therapeutics, Alameda, CA (M.J.H., B.L.O.).
John J P KasteleinDepartment of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, The Netherlands (R.F.O., E.S.G.S., J.J.P.K.).ORCID 0000-0003-2018-4532

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

There is overwhelming clinical and genetic evidence supporting the concept that low-density-lipoprotein cholesterol should be as low as possible for as long as possible in patients at very high cardiovascular risk. Despite the wide availability of effective lipid-lowering therapies, the majority of patients still fail to reach guideline-based lipid goals. Advances in novel approaches targeting PCSK9 (proprotein convertase subtilisin/kexin type 9) through small-interfering RNA and genome editing hold the potential to bridge this gap, by offering long-acting alternatives, which may overcome adherence and other challenges in the current chronic care model. In this review, we discuss the history of targeting PCSK9 with the use of mRNA and small-interfering ribonucleic acid. We also shed light on targeting PCSK9 with genome editing, including discussion of the VERVE-101 clustered regularly interspaced short palindromic repeats-base editing medicine currently being evaluated in a clinical trial and others in development.

Indexed as

Gene EditingProprotein Convertase 9Cholesterol, LDLHumansRNA, Small InterferingCholesterol, LDLPCSK9 protein, humanProprotein Convertase 9RNA, Small Interferingapolipoproteincardiologycoronary artery diseasemutationsubtilisin

Identifiers

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.