Evidence map›Paper›PMID 30622323›Full record

ArticleCancer gene therapy2019

Schwannoma gene therapy by adeno-associated virus delivery of the pore-forming protein Gasdermin-D.

Sherif G Ahmed, Ahmed Abdelanabi, Mohamed Doha, Gary J Brenner

Erratum issuedAbstract read
PubMed Publisher
In one paragraph

Article in Cancer gene therapy, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 23 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
23citing papers in PubMed, 1 pooled it
3.0field-weighted citation impact, top 9% 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

23 citing papers in PubMed, 1 synthesis or guideline pooled it, 28 citations in OpenAlex.

  1. Pooled it
  2. Deletion of themBio · 2026
    Article
  3. Review
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  8. Culture of attenuatedHuman vaccines & immunotherapeutics · 2023
    Article
  9. Review
  10. Gene replacement therapy in a schwannoma mouse model of neurofibromatosis type 2.Molecular therapy. Methods & clinical development · 2022
    Article
  11. Intratumoral injection of schwannoma with attenuatedProceedings of the National Academy of Sciences of the United States of America · 2022
    Article
  12. Review
  13. Article
  14. Article
  15. Article
  16. Article
  17. Article
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

4 authors at 2 institutions in 1 country.

Sherif G AhmedDepartment of Anesthesiology, Critical Care, and Pain Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, 02114, USA.
Ahmed AbdelanabiDepartment of Anesthesiology, Critical Care, and Pain Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, 02114, USA.
Mohamed DohaDepartment of Anesthesiology, Critical Care, and Pain Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, 02114, USA.
Gary J BrennerDepartment of Anesthesiology, Critical Care, and Pain Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, 02114, USA. gjbrenner@mgh.harvard.edu.
Massachusetts General Hospital · USHarvard University · US

Funding

Mechanisms of Caspase-1 Mediated Schwannoma RegressionR01NS081146 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI BRENNER, GARY JAY · 2012 to 2016
$1.8M
Translation of AAV P0 ICE schwannoma gene therapy to clinical TrialsR21NS088013 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI BRENNER, GARY JAY · 2015 to 2016
$846k
NINDS NIH HHS R01 NS081146NINDS NIH HHS R21 NS088013
6 · The paper itself

Abstract

Schwannomas are peripheral nerve sheath tumors associated with three genetically distinct disease entities, namely sporadic schwannoma, neurofibromatosis type-2, and schwannomatosis. Schwannomas are associated with severe disability and in cases lead to death. The primary treatment is operative resection that itself can cause neurologic damage and is at times contra-indicated due to tumor location. Given their homogenous Schwann-lineage cellular composition, schwannomas are appealing targets for gene therapy. In the present study, we have generated an adeno-associated serotype 1 virus (AAV1)-based vector delivering N-terminal of the pyroptotic gene Gasdermin-D; (GSDMDNterm) under the control of the Schwann-cell specific promoter, P0. we have demonstrated that AAV1-P0-GSDMDNterm injection into intra-sciatic schwannomas reduces the growth of these tumors and resolves tumor-associated pain without causing neurologic damage. This AAV1-P0-GSDMDNterm vector holds promise for clinical treatment of schwannomas via direct intra-tumoral injection.

Indexed as

Genetic TherapyAnimalsBiomarkersCell Line, TumorCell SurvivalDependovirusDisease Models, AnimalGasderminsGene ExpressionGenetic VectorsGene Transfer TechniquesHumansInjections, IntralesionalIntracellular Signaling Peptides and ProteinsMaleMiceBiomarkersGasderminsGSDMD protein, humanIntracellular Signaling Peptides and ProteinsPhosphate-Binding Proteins

Identifiers

PMID30622323
OpenAlexW2908857457

What OpenQuestion holds

Textmetadata
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.