Evidence map›Paper›PMID 31906492›Full record

ReviewCells2020

Context-Dependent Strategies for Enhanced Genome Editing of Genodermatoses.

Oliver Patrick March, Thomas Kocher, Ulrich Koller

Open access · goldAbstract readReview
In one paragraph

Review in Cells, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed
2.3field-weighted citation impact, top 11% 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

18 citing papers in PubMed, 35 citations in OpenAlex.

  1. Article
  2. Review
  3. Emerging Gene Therapeutics for Epidermolysis Bullosa under Development.International journal of molecular sciences · 2024
    Review
  4. Gene therapy advances shine the spotlight on epidermolysis bullosa, bringing hope to patients.Molecular therapy : the journal of the American Society of Gene Therapy · 2023
    Article
  5. Article
  6. Paired nicking-mediated COL17A1 reframing for junctional epidermolysis bullosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2022
    Article
  7. Detection of Novel Biallelic Causative Variants inDiagnostics (Basel, Switzerland) · 2022
    Article
  8. Article
  9. Review
  10. A non-viral and selection-freeMolecular therapy. Nucleic acids · 2021
    Article
  11. Review
  12. Article
  13. Signatures of Dermal Fibroblasts from RDEB Pediatric Patients.International journal of molecular sciences · 2021
    Article
  14. Review
  15. Review
  16. Article
  17. Epidermolysis bullosa.Nature reviews. Disease primers · 2020
    Review
  18. 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.

Oliver Patrick MarchEB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, 5020 Salzburg, Austria.ORCID 0000-0002-1140-310X
Thomas KocherEB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, 5020 Salzburg, Austria.ORCID 0000-0002-3424-3049
Ulrich KollerEB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, 5020 Salzburg, Austria.ORCID 0000-0002-6285-1789
Paracelsus Medical University · AT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The skin provides direct protection to the human body from assault by the harsh external environment. The crucial function of this organ is significantly disrupted in genodermatoses patients. Genodermatoses comprise a heterogeneous group of largely monogenetic skin disorders, typically involving mutations in genes encoding structural proteins. Therapeutic options for this debilitating group of diseases, including epidermolysis bullosa, primarily consist of wound management. Genome editing approaches co-opt double-strand break repair pathways to introduce desired sequence alterations at specific loci. Rapid advances in genome editing technologies have the potential to propel novel genetic therapies into the clinic. However, the associated phenotypes of many mutations may be treated via several genome editing strategies. Therefore, for potential clinical applications, implementation of efficient approaches based upon mutation, gene and disease context is necessary. Here, we describe current genome editing approaches for the treatment of genodermatoses, along with a discussion of the optimal strategy for each genetic context, in order to achieve enhanced genome editing approaches.

Indexed as

Gene EditingExonsGenetic TherapyHomologous RecombinationHumansSkinSkin Diseasesdesigner nucleasesDNA repair pathwaysepidermolysis bullosagenodermatosesgenome editingkeratinopathies

Identifiers

PMID31906492
PMCPMC7016731
OpenAlexW2997832903

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.