Evidence map›Paper›PMID 37641403›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2023

AAV9-mediated SH3TC2 gene replacement therapy targeted to Schwann cells for the treatment of CMT4C.

Elena Georgiou, Alexia Kagiava, Irene Sargiannidou, Natasa Schiza, Marina Stavrou, Jan Richter, Christina Tryfonos, Amanda Heslegrave, Henrik Zetterberg, Christina Christodoulou and 1 more

Open access · bronzeAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed
2.4field-weighted citation impact, top 11% 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

14 citing papers in PubMed, 16 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Charcot-Marie-Tooth disease and related neuropathies.Nature reviews. Disease primers · 2026
    Review
  5. Article
  6. Review
  7. Observational
  8. Review
  9. A dose escalation and safety study of AAVrh10-mediated Schwann cell-targeted gene therapy for CMT1X.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2025
    Article
  10. Article
  11. Review
  12. Review
  13. Article
  14. 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

11 authors at 3 institutions in 3 countries.

Elena GeorgiouNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Alexia KagiavaNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Irene SargiannidouNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Natasa SchizaNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Marina StavrouNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Jan RichterMolecular Virology Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Christina TryfonosMolecular Virology Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Amanda HeslegraveDepartment of Neurodegenerative Disease, UCL Institute of Neurology, London, UK; UK Dementia Research Institute at UCL, London, UK.
Henrik ZetterbergDepartment of Neurodegenerative Disease, UCL Institute of Neurology, London, UK; UK Dementia Research Institute at UCL, London, UK; Department of Psychiatry and Neurochemistry, Institute of Neuroscience and Physiology, the Sahlgrenska Academy at the University of Gothenburg, Mölndal, Sweden; Clinical Neurochemistry Laboratory, Sahlgrenska University Hospital, Mölndal, Sweden; Hong Kong Center for Neurodegenerative Diseases, Clear Water Bay, Hong Kong, China; Wisconsin Alzheimer's Disease Research Center, University of Wisconsin School of Medicine and Public Health, University of Wisconsin-Madison, Madison, WI, USA.
Christina ChristodoulouMolecular Virology Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Kleopas A KleopaNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus; Center for Neuromuscular Disorders, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus. Electronic address: kleopa@cing.ac.cy.
Cyprus Institute of Neurology and Genetics · CYSahlgrenska University Hospital · SEUK Dementia Research Institute · GB

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Type 4C Charcot-Marie-Tooth (CMT4C) demyelinating neuropathy is caused by autosomal recessive SH3TC2 gene mutations. SH3TC2 is highly expressed in myelinating Schwann cells. CMT4C is a childhood-onset progressive disease without effective treatment. Here, we generated a gene therapy for CMT4C mediated by an adeno-associated viral 9 vector (AAV9) to deliver the human SH3TC2 gene in the Sh3tc2

Indexed as

Charcot-Marie-Tooth DiseaseGenetic TherapyIntracellular Signaling Peptides and ProteinsAnimalsHumansMiceMutationSchwann CellsIntracellular Signaling Peptides and ProteinsSH3TC2 protein, humanAAV9Charcot-Marie-Tooth 4Cgene therapyperipheral neuropathySchwann cells

Identifiers

PMID37641403
PMCPMC10638072
OpenAlexW4386227215

What OpenQuestion 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 OpenQuestion graph.