Evidence map›Paper›PMID 26073463›Full record

ReviewTranslational research : the journal of laboratory and clinical medicine2016

Gene editing toward the use of autologous therapies in recessive dystrophic epidermolysis bullosa.

Christopher Perdoni, Mark J Osborn, Jakub Tolar

Abstract readReview
In one paragraph

Review in Translational research : the journal of laboratory and clinical medicine, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
1.6field-weighted citation impact, top 18% 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

8 citing papers in PubMed, 20 citations in OpenAlex.

  1. Review
  2. Review
  3. Article
  4. Pathogenetic Therapy of Epidermolysis Bullosa: Current State and Prospects.Bulletin of experimental biology and medicine · 2021
    Review
  5. Review
  6. Review
  7. Article
  8. Genome-editing Technologies for Gene and Cell Therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2016
    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

3 authors at 1 institution in 1 country.

Christopher PerdoniStem Cell Institute, University of Minnesota, Minneapolis, Minn; Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minn.
Mark J OsbornStem Cell Institute, University of Minnesota, Minneapolis, Minn; Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minn.
Jakub TolarStem Cell Institute, University of Minnesota, Minneapolis, Minn; Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minn. Electronic address: morga336@umn.edu.
University of Minnesota Medical Center · US

Funding

University of Minnesota Clinical and Translational Science Institute (UMN CTSI)UL1TR000114 · NCATS · UNIVERSITY OF MINNESOTA · PI BLAZAR, BRUCE R · 2012 to 2015
$35.0M
Skin-targeted Cell Therapy for Recessive Dystrophic Epidermolysis BullosaR01AR063070 · NIAMS · UNIVERSITY OF MINNESOTA · PI Mark J Osborn, Jakub Tolar · 2013 to 2026
$4.5M
Testing the Therapeutic Potential of iPS Cells for Inherited Skin DiseasesR01AR059947 · NIAMS · UNIVERSITY OF COLORADO DENVER · PI ROOP, DENNIS · 2012 to 2017
$2.1M
NCATS NIH HHS UL1 TR000114NCATS NIH HHS UL1TR000114NIAMS NIH HHS R01 AR059947NIAMS NIH HHS R01AR059947NIAMS NIH HHS R01 AR063070
6 · The paper itself

Abstract

Recessive dystrophic epidermolysis bullosa (RDEB) is a disease caused by mutations in the COL7A1 gene that result in absent or dysfunctional type VII collagen protein production. Clinically, RDEB manifests as early and severe chronic cutaneous blistering, damage to internal epithelium, an increased risk for squamous cell carcinoma, and an overall reduced life expectancy. Recent localized and systemic treatments have shown promise for lessening the disease severity in RDEB, but the concept of ex vivo therapy would allow a patient's own cells to be engineered to express functional type VII collagen. Here, we review gene delivery and editing platforms and their application toward the development of next-generation treatments designed to correct the causative genetic defects of RDEB.

Indexed as

Genetic EngineeringCollagen Type VIIEpidermolysis Bullosa DystrophicaGenetic Predisposition to DiseaseGenetic TherapyHumansCOL7A1 protein, humanCollagen Type VII

Identifiers

PMID26073463
PMCPMC4662628
OpenAlexW2217626770

What OpenQuestion holds

Textmetadata
LicenceTDM
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.