Evidence map›Paper›PMID 38103141›Full record

ArticleMolecular diagnosis & therapy2024

Viral Vector-Based Gene Therapy for Epilepsy: What Does the Future Hold?

Barbara Bettegazzi, Stefano Cattaneo, Michele Simonato, Silvia Zucchini, Marie Soukupova

Open access · hybridAbstract read
In one paragraph

Article in Molecular diagnosis & therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
3.8field-weighted citation impact, top 6% 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, 12 citations in OpenAlex.

  1. Review
  2. CRISPR-Based Gene Therapy for Brain Disease.Molecular neurobiology · 2026
    Review
  3. Article
  4. Article
  5. Review
  6. Review
  7. Article
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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

5 authors at 2 institutions in 1 country.

Barbara Bettegazzi *University Vita-Salute San Raffaele, Milan, Italy.
Stefano Cattaneo *Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Michele SimonatoDivision of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Silvia ZucchiniDepartment of Neuroscience and Rehabilitation, University of Ferrara, via Fossato di Mortara 70, 44121, Ferrara, Italy. silvia.zucchini@unife.it.ORCID 0000-0001-9010-6447
Marie SoukupovaDepartment of Neuroscience and Rehabilitation, University of Ferrara, via Fossato di Mortara 70, 44121, Ferrara, Italy.
Vita-Salute San Raffaele University · ITUniversity of Ferrara · IT

Funding

Horizon 2020 Framework Programme 964712
6 · The paper itself

Abstract

In recent years, many pre-clinical studies have tested gene therapy approaches as possible treatments for epilepsy, following the idea that they may provide an alternative to conventional pharmacological and surgical options. Multiple gene therapy approaches have been developed, including those based on anti-sense oligonucleotides, RNA interference, and viral vectors. In this opinion article, we focus on translational issues related to viral vector-mediated gene therapy for epilepsy. Research has advanced dramatically in addressing issues like viral vector optimization, target identification, strategies of gene expression, editing or regulation, and safety. Some of these pre-clinically validated potential gene therapies are now being tested in clinical trials, in patients with genetic or focal forms of drug-resistant epilepsy. Here, we discuss the ongoing translational research and the advancements that are needed and expected in the near future. We then describe the clinical trials in the pipeline and the further challenges that will need to be addressed at the clinical and economic levels. Our optimistic view is that all these issues and challenges can be overcome, and that gene therapy approaches for epilepsy will soon become a clinical reality.

Indexed as

EpilepsyGenetic TherapyGenetic VectorsHumansOligonucleotides, AntisenseOligonucleotides, Antisense

Identifiers

PMID38103141
PMCPMC10786988
OpenAlexW4389828786

What OpenQuestion 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 OpenQuestion graph.