Evidence map›Paper›PMID 39429724›Full record

ArticleMolecular therapy. Methods & clinical development2024

A novel AAV9-dual microRNA-vector targeting

Stéphane J Baudouin, April R Giles, Nick Pearson, Severine Deforges, Chenxia He, Céline Boileau, Nicolas Partouche, Andreas Borta, Justine Gautron, Morgane Wartel and 12 more

Abstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing 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

10 citing papers in PubMed.

  1. Review
  2. Article
  3. Article
  4. Genetic medicines for epilepsy: unlocking new avenues for seizure control.Frontiers in bioengineering and biotechnology · 2026
    Review
  5. Article
  6. Article
  7. Targeting the trunk of multi-root common epilepsy with gene therapy.Molecular therapy. Methods & clinical development · 2025
    Article
  8. State-of-the-art gene therapy in epilepsy.Current opinion in neurology · 2025
    Review
  9. Review
  10. 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

22 authors.

Stéphane J BaudouinuniQure (Corlieve Therapeutics AG), 4052 Basel, Switzerland.
April R GilesREGENXBIO Inc., Rockville, MD 20850, USA.
Nick PearsonuniQure (Corlieve Therapeutics AG), 4052 Basel, Switzerland.
Severine DeforgesInstitute for Neuroscience, 33000 Bordeaux, France.
Chenxia HeuniQure (Corlieve Therapeutics AG), 4052 Basel, Switzerland.
Céline BoileauINSERM, INMED, Aix-Marseille University, 13009 Marseille, France.
Nicolas PartoucheuniQure (Corlieve Therapeutics AG), 4052 Basel, Switzerland.
Andreas BortauniQure (Corlieve Therapeutics AG), 4052 Basel, Switzerland.
Justine GautronInstitute for Neuroscience, 33000 Bordeaux, France.
Morgane WarteluniQure biopharma B.V., 1105BP Amsterdam, the Netherlands.
Irena BočkajuniQure biopharma B.V., 1105BP Amsterdam, the Netherlands.
Didier ScavardaAPHM, INSERM, Aix-Marseille University, Timone Hospital, Pediatric Neurosurgery, 13005 Marseille, France.
Fabrice BartolomeiAPHM, INSERM, Aix-Marseille University, INS, Timone Hospital, Epileptology Department, 13005 Marseille, France.
Guillaume PenchetPellegrin Hospital, Neurosurgery Department, CHU, 33000 Bordeaux, France.
Jérôme AupyPellegrin Hospital, Neurosurgery Department, CHU, 33000 Bordeaux, France.
Jennifer SimsIntegrated Biologix GmbH, 4051 Basel, Switzerland.
Jared SmithREGENXBIO Inc., Rockville, MD 20850, USA.
Andrew MercerREGENXBIO Inc., Rockville, MD 20850, USA.
Olivier DanosREGENXBIO Inc., Rockville, MD 20850, USA.
Christophe MulleInstitute for Neuroscience, 33000 Bordeaux, France.
Valérie CrépelINSERM, INMED, Aix-Marseille University, 13009 Marseille, France.
Richard PorteruniQure (Corlieve Therapeutics AG), 4052 Basel, Switzerland.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Mesial temporal lobe epilepsy (mTLE) is the most prevalent type of epilepsy in adults. First and subsequent generations of anti-epileptic therapy regimens fail to decrease seizures in a large number of patients suffering from mTLE, leaving surgical ablation of part of the hippocampus as the only therapeutic option to potentially reach seizure freedom. GluK2 has recently been identified as a promising target for the treatment of mTLE using gene therapy. Here, we engineered an adeno-associated virus serotype 9 vector expressing a cluster of two synthetic microRNAs (miRNAs), expressed from the human synapsin promoter, that target

Indexed as

AAV9epilepsygene therapyGRIK2microRNA

Identifiers

PMID39429724
PMCPMC11489344

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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.