Evidence map›Paper›PMID 31194088›Full record

ArticleMolecular therapy. Methods & clinical development2019

In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver.

Jolanda M Liefhebber, Raygene Martier, Tom Van der Zon, Sonay Keskin, Angelina Huseinovic, Jacek Lubelski, Bas Blits, Harald Petry, Pavlina Konstantinova

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
0.4field-weighted citation impact, top 31% 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

2 citing papers in PubMed, 7 citations in OpenAlex.

  1. Article
  2. 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

9 authors at 2 institutions in 1 country.

Jolanda M LiefhebberDepartment of Research & Development, uniQure N.V., 1105BP Amsterdam, the Netherlands.
Raygene MartierDepartment of Research & Development, uniQure N.V., 1105BP Amsterdam, the Netherlands.
Tom Van der ZonDepartment of Research & Development, uniQure N.V., 1105BP Amsterdam, the Netherlands.
Sonay KeskinDepartment of Research & Development, uniQure N.V., 1105BP Amsterdam, the Netherlands.
Angelina HuseinovicDepartment of Research & Development, uniQure N.V., 1105BP Amsterdam, the Netherlands.
Jacek LubelskiDepartment of Research & Development, uniQure N.V., 1105BP Amsterdam, the Netherlands.
Bas BlitsDepartment of Research & Development, uniQure N.V., 1105BP Amsterdam, the Netherlands.
Harald PetryDepartment of Research & Development, uniQure N.V., 1105BP Amsterdam, the Netherlands.
Pavlina KonstantinovaDepartment of Research & Development, uniQure N.V., 1105BP Amsterdam, the Netherlands.
UniQure (Netherlands) · NLAmsterdam University Medical Centers · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Gene therapy is being developed for the treatment of inherited diseases, whereby a therapeutic gene is continuously expressed in patients after delivery via viral vectors such as adeno-associated virus (AAV). Depending on the transgene, there could be a limited therapeutic window, and regulating timing and levels of transgene expression is advantageous. To control transgene transcription, the regulatory system GeneSwitch (GS) was evaluated in detail both

Indexed as

AAV5 gene therapymifepristone-inducible GeneSwitch systemregulated gene expression

Identifiers

PMID31194088
PMCPMC6551379
OpenAlexW2946313225

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.