Evidence map›Paper›PMID 23140466›Full record

ArticleHuman gene therapy2013

Regression of schwannomas induced by adeno-associated virus-mediated delivery of caspase-1.

Shilpa Prabhakar, Mehran Taherian, Davide Gianni, Thomas J Conlon, Giulia Fulci, Jillian Brockmann, Anat Stemmer-Rachamimov, Miguel Sena-Esteves, Xandra O Breakefield, Gary J Brenner

Abstract read
In one paragraph

Article in Human gene therapy, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. 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
–field-weighted citation impact, top 84% 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, 27 citations in OpenAlex.

  1. Pooled it
  2. Review
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  8. Review
  9. Gene replacement therapy in a schwannoma mouse model of neurofibromatosis type 2.Molecular therapy. Methods & clinical development · 2022
    Article
  10. A Critical Overview of Targeted Therapies for Vestibular Schwannoma.International journal of molecular sciences · 2022
    Review
  11. Article
  12. Review
  13. Article
  14. Transcriptomic signature of painful human neurofibromatosis type 2 schwannomas.Annals of clinical and translational neurology · 2021
    Article
  15. Review
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  17. Article
  18. 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

10 authors at 3 institutions in 1 country.

Shilpa PrabhakarNeuroscience Center, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital, Boston, MA 02114, USA.
Mehran Taherian
Davide Gianni
Thomas J Conlon
Giulia Fulci
Jillian Brockmann
Anat Stemmer-Rachamimov
Miguel Sena-Esteves
Xandra O Breakefield
Gary J Brenner
Massachusetts General Hospital · USUniversity of Massachusetts Chan Medical School · USUniversity of Florida · US

Funding

Vector Query and Therapy For TSC and NF2 LesionsP01NS024279 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI KWIATKOWSKI, DAVID J. · 1987 to 2012
$14.7M
Mechanisms of Caspase-1 Mediated Schwannoma RegressionR01NS081146 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI BRENNER, GARY JAY · 2012 to 2016
$1.8M
NINDS NIH HHS NS24279NINDS NIH HHS R01 NS081146
6 · The paper itself

Abstract

Schwannomas are tumors formed by proliferation of dedifferentiated Schwann cells. Patients with neurofibromatosis 2 (NF2) and schwannomatosis develop multiple schwannomas in peripheral and cranial nerves. Although benign, these tumors can cause extreme pain and compromise sensory/motor functions, including hearing and vision. At present, surgical resection is the main treatment modality, but it can be problematic because of tumor inaccessibility and risk of nerve damage. We have explored gene therapy for schwannomas, using a model in which immortalized human NF2 schwannoma cells expressing a fluorescent protein and luciferase are implanted in the sciatic nerve of nude mice. Direct injection of an adeno-associated virus (AAV) serotype 1 vector encoding caspase-1 (ICE) under the Schwann-cell specific promoter, P0, leads to regression of these tumors with essentially no vector-mediated neuropathology, and no changes in sensory or motor function. In a related NF2 xenograft model designed to cause measurable pain behavior, the same gene therapy leads to tumor regression and concordant resolution of tumor-associated pain. This AAV1-P0-ICE vector holds promise for clinical treatment of schwannomas by direct intratumoral injection to achieve reduction in tumor size and normalization of neuronal function.

Indexed as

AnimalsCaspase 1DependovirusGenetic TherapyGenetic VectorsGreen Fluorescent ProteinsHEK293 CellsHumansMiceMice, Inbred C57BLMice, NudeNeurilemmomaNeurofibromatosis 2PlasmidsPromoter Regions, GeneticPsychomotor PerformanceCaspase 1Green Fluorescent Proteins

Identifiers

PMID23140466
PMCPMC3581065
OpenAlexW2144540418

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.