Evidence map›Paper›PMID 32178539›Full record

ReviewExpert opinion on biological therapy2020

Leading edge: emerging drug, cell, and gene therapies for junctional epidermolysis bullosa.

Allison R Keith, Kirk Twaroski, Christen L Ebens, Jakub Tolar

Open access · greenAbstract readReview
In one paragraph

Review in Expert opinion on biological therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
1.7field-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

11 citing papers in PubMed, 19 citations in OpenAlex.

  1. Review
  2. Review
  3. Article
  4. Article
  5. Review
  6. Article
  7. Development and clinical translation ofComputational and structural biotechnology journal · 2022
    Review
  8. Article
  9. Review
  10. Review
  11. Article
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

4 authors at 1 institution in 1 country.

Allison R KeithDepartment of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota , Minneapolis, MN, USA.
Kirk TwaroskiDepartment of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota , Minneapolis, MN, USA.
Christen L EbensDepartment of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota , Minneapolis, MN, USA.ORCID 0000-0003-2430-911X
Jakub TolarDepartment of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota , Minneapolis, MN, USA.
University of Minnesota · US

Funding

University of Minnesota Clinical and Translational Science Institute (UMN CTSI)UL1TR002494 · NCATS · UNIVERSITY OF MINNESOTA · PI BLAZAR, BRUCE R, WEISDORF, DANIEL J · 2018 to 2022
$34.9M
Institutional Career Development CoreKL2TR002492 · NCATS · UNIVERSITY OF MINNESOTA · PI INGBAR, DAVID H · 2018 to 2022
$6.3M
NCATS NIH HHS KL2 TR002492NCATS NIH HHS UL1 TR002494
6 · The paper itself

Abstract

introductionJunctional epidermolysis bullosa (JEB) is a rare inherited genetic disorder with limited treatments beyond palliative care. A major hallmark of JEB is skin blistering caused by functional loss or complete absence of major structural proteins of the skin. Impaired wound healing in patients with JEB gives rise to chronic cutaneous ulcers that require daily care. Wound care and infection control are the current standard of care for this patient population. AREAS COVERED: This review covers research and clinical implementation of emerging drug, cell, and gene therapies for JEB. Current clinical trials use topical drug delivery to manipulate the inflammation and re-epithelialization phases of wound healing or promote premature stop codon readthrough to accelerate chronic wound closure. Allogeneic cell therapies for JEB have been largely unsuccessful, with autologous skin grafting emerging as a reliable method of resolving the cutaneous manifestations of JEB. Genetic correction and transplant of autologous keratinocytes have demonstrated persistent amelioration of chronic wounds in a subset of patients. EXPERT OPINION: Emerging therapies address the cutaneous symptoms of JEB but are unable to attend to systemic manifestations of the disease. Investigations into the molecular mechanism(s) underpinning the failure of systemic allogeneic cell therapies are necessary to expand the range of effective JEB therapies.

Indexed as

Cell Adhesion MoleculesDependovirusEpidermolysis Bullosa, JunctionalGenetic TherapyGenetic VectorsHematopoietic Stem Cell TransplantationHumansKalininSkin TransplantationTriterpenesCell Adhesion MoleculesKalininTriterpenesBasement membrane zonecell therapyclinical trialsdrug therapyextracellular matrixgene therapyhematopoietic cell transplantjunctional epidermolysis bullosakeratinocyteswound healing

Identifiers

PMID32178539
PMCPMC7392816
OpenAlexW3012343987

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.