Evidence mapPaperPMID 40600248Full record

ReviewEuropean heart journal2025

Gene therapy and genome editing for lipoprotein disorders.

Chen Gurevitz, Archna Bajaj, Amit V Khera, Ron Do, Heribert Schunkert, Kiran Musunuru, Robert S Rosenson

Abstract readReview
In one paragraph

Review in European heart journal, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.

0numbers the graph read from it
0cells of the map it votes in
15citing 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

15 citing papers in PubMed.

  1. Review
  2. Review
  3. Review
  4. Review
  5. Old and New Lines of Therapy Targeting Lipoprotein(a).Current atherosclerosis reports · 2026
    Review
  6. Review
  7. Review
  8. Genetic factors contributing to atherosclerosis.Current opinion in cardiology · 2026
    Review
  9. Review
  10. Review
  11. Article
  12. Review
  13. Review
  14. Review
  15. Review
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.

Chen GurevitzMetabolism and Lipids Program, Mount Sinai Fuster Heart Hospital, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, Box 1030, New York, NY 10029, USA.ORCID 0000-0003-2632-6669
Archna BajajDivision of Translational Medicine and Human Genetics, University of Pennsylvania, Philadelphia, PA, USA.ORCID 0000-0001-7397-6520
Amit V KheraDepartment of Medicine, Division of Cardiovascular Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.
Ron DoDepartment of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA.ORCID 0000-0002-3144-3627
Heribert SchunkertDepartment of Cardiology, Deutsches Herzzentrum München, Technische Universität München, Munich, Germany.ORCID 0000-0001-6428-3001
Kiran MusunuruDivision of Cardiovascular Medicine, Cardiovascular Institute, University of Pennsylvania, Philadelphia, PA, USA.ORCID 0000-0003-3298-0368
Robert S RosensonMetabolism and Lipids Program, Mount Sinai Fuster Heart Hospital, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, Box 1030, New York, NY 10029, USA.ORCID 0000-0002-5599-0633

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Genetic factors play a critical role in the development of lipoprotein disorders, which significantly contribute to atherosclerotic cardiovascular disease (ASCVD). Traditional management of these conditions has relied on lipid-lowering therapies, which require lifelong adherence. Recent advancements in gene addition and editing technologies offer novel and potentially transformative approaches for treating lipoprotein disorders by targeting the relevant genetic pathways for each disease. This review revisits major monogenic and polygenic disorders of lipoprotein metabolism, including familial hypercholesterolemia, elevated lipoprotein(a), and familial chylomicronemia syndrome, and discusses the genetic-based therapies for management. RNA-based, gene addition and gene editing therapies, including Clustered Regularly Interspaced Short Palindromic Repeats, base editing and interventions whereby, are highlighted for their potential to provide durable treatments which overcome the adherence challenge. Integration of machine learning for risk prediction and the use of polygenic risk scores to enhance risk stratification further demonstrate the promise of personalized approaches, and overall potential for gene-based treatments to revolutionize ASCVD prevention and management.

Indexed as

Gene EditingGenetic TherapyAtherosclerosisHumansHyperlipoproteinemia Type IILipoprotein(a)Lipoprotein(a)Cholesterol lowering therapyGene editingLipoprotein disordersMachine learningMonogenic disordersPolygenic risk scores

Identifiers

PMID40600248
PMCPMC12448413

What Socratic holds

Textmetadata
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 Socratic graph.