Evidence map›Paper›PMID 39684857›Full record

ReviewInternational journal of molecular sciences2024

Unveiling the Future of Cardiac Care: A Review of Gene Therapy in Cardiomyopathies.

Damiano Venturiello, Pier Giorgio Tiberi, Francesco Perulli, Giulia Nardoianni, Leonardo Guida, Carlo Barsali, Carlo Terrone, Alessandro Cianca, Camilla Lustri, Matteo Sclafani and 3 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in International journal of molecular sciences, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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

6 citing papers in PubMed.

  1. Review
  2. Article
  3. Review
  4. Review
  5. Review
  6. 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

13 authors.

Damiano VenturielloCardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy.ORCID 0009-0000-0874-9735
Pier Giorgio TiberiCardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy.
Francesco PerulliCardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy.
Giulia NardoianniCardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy.ORCID 0000-0001-6861-6870
Leonardo GuidaCardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy.
Carlo BarsaliCardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy.ORCID 0009-0001-5216-212X
Carlo TerroneCardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy.
Alessandro CiancaCardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy.
Camilla LustriCardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy.
Matteo SclafaniCardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy.
Giacomo TiniCardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy.ORCID 0000-0001-8520-152X
Emanuele BarbatoCardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy.
Beatrice MusumeciCardiology, Clinical and Molecular Medicine Department, Sapienza University of Rome, 00189 Rome, Italy.ORCID 0000-0003-4872-7981

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

For years, the treatment of many cardiomyopathies has been solely focused on symptom management. However, cardiomyopathies have a genetic substrate, and directing therapy towards the pathophysiology rather than the epiphenomenon of the disease may be a winning strategy. Gene therapy involves the insertion of genes or the modification of existing ones and their regulatory elements through strategies like gene replacement and gene editing. Recently, gene therapy for cardiac amyloidosis and Duchenne muscular dystrophy has received approval, and important clinical trials are currently evaluating gene therapy methods for rare heart diseases like Friedreich's Ataxia, Danon disease, Fabry disease, and Pompe Disease. Furthermore, favorable results have been noted in animal studies receiving gene therapy for hypertrophic, dilated, and arrhythmogenic cardiomyopathy. This review discusses gene therapy methods, ongoing clinical trials, and future goals in this area.

Indexed as

CardiomyopathiesGenetic TherapyAnimalsGene EditingHumansadeno-associated virus (AAV)cardiomyopathiesclinical trials in cardiomyopathiesCRISPR/Cas9gene editinggene replacementgene therapytransgene expressionvector delivery

Identifiers

PMID39684857
PMCPMC11642060

What OpenQuestion holds

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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.