Evidence map›Paper›PMID 42120546›Full record

ArticleGene therapy2026

A dose-escalation and safety gene therapy study in a model of CMT4C neuropathy.

Elena Georgiou, Alexia Kagiava, Andreas Hentschel, Irene Sargiannidou, Revekka Papacharalampous, Marina Stavrou, Christina Tryfonos, Jan Richter, Andreas Roos, Kleopas A Kleopa

Abstract read
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In one paragraph

Article in Gene therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

10 authors.

Elena GeorgiouNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus. elenag@cing.ac.cy.ORCID http://orcid.org/0000-0003-4207-7973
Alexia KagiavaNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.ORCID http://orcid.org/0000-0002-3903-1299
Andreas HentschelLeibniz-Institute for Analytical Sciences -ISAS- e.V., Dortmund, Germany.
Irene SargiannidouNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Revekka PapacharalampousNeuropathology Laboratory, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Marina StavrouNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.ORCID http://orcid.org/0000-0002-3637-6523
Christina TryfonosMolecular Virology Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Jan RichterMolecular Virology Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Andreas RoosDepartment of Pediatric Neurology, Centre for Neuromuscular Disorders, University Duisburg-Essen, Essen, Germany.
Kleopas A KleopaNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.ORCID http://orcid.org/0000-0002-4103-8094

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Charcot-Marie-Tooth disease type 4C is a demyelinating neuropathy caused by loss of function mutations in the SH3TC2 gene, that is highly expressed in myelinating Schwann cells. We generated and tested a clinical stage vector with a minimal human MPZ promoter driving expression of SH3TC2. Groups of 1-month old Sh3tc2

Identifiers

PMID42120546

What OpenQuestion holds

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Registered trials

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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.