Evidence map›Paper›PMID 34110606›Full record

ReviewDermatology and therapy2021

Clinical Perspectives of Gene-Targeted Therapies for Epidermolysis Bullosa.

Tobias Welponer, Christine Prodinger, Josefina Pinon-Hofbauer, Arno Hintersteininger, Hannelore Breitenbach-Koller, Johann W Bauer, Martin Laimer

Open access · goldAbstract readReview
In one paragraph

Review in Dermatology and therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed
2.1field-weighted citation impact, top 14% 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

14 citing papers in PubMed, 25 citations in OpenAlex.

  1. Review
  2. Article
  3. RiboScreenBiomedicines · 2026
    Review
  4. Review
  5. Review
  6. Review
  7. Emerging Gene Therapeutics for Epidermolysis Bullosa under Development.International journal of molecular sciences · 2024
    Review
  8. Article
  9. Article
  10. Article
  11. Stairways to Advanced Therapies for Epidermolysis Bullosa.Cold Spring Harbor perspectives in biology · 2023
    Review
  12. Stem Cell Therapies for Epidermolysis Bullosa Treatment.Bioengineering (Basel, Switzerland) · 2023
    Review
  13. Review
  14. Development and clinical translation ofComputational and structural biotechnology journal · 2022
    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

7 authors at 3 institutions in 1 country.

Tobias Welponer *Department of Dermatology and Allergology and EB House Austria, University Hospital of the Paracelsus Medical University, Salzburg, Austria.
Christine Prodinger *Department of Dermatology and Allergology and EB House Austria, University Hospital of the Paracelsus Medical University, Salzburg, Austria.
Josefina Pinon-HofbauerDepartment of Dermatology and Allergology and EB House Austria, University Hospital of the Paracelsus Medical University, Salzburg, Austria.
Arno HintersteiningerDepartment of Dermatology and Allergology, University Hospital of the Paracelsus Medical University, Salzburg, Austria.
Hannelore Breitenbach-KollerDepartment of Biosciences, Paris Lodron University of Salzburg, Salzburg, Austria.
Johann W BauerDepartment of Dermatology and Allergology and EB House Austria, University Hospital of the Paracelsus Medical University, Salzburg, Austria.
Martin LaimerDepartment of Dermatology and Allergology and EB House Austria, University Hospital of the Paracelsus Medical University, Salzburg, Austria. m.laimer@salk.at.
Paracelsus Medical University · ATEB House Austria · ATUniversity of Salzburg · AT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

New insights into molecular genetics and pathomechanisms in epidermolysis bullosa (EB), methodological and technological advances in molecular biology as well as designated funding initiatives and facilitated approval procedures for orphan drugs have boosted translational research perspectives for this devastating disease. This is echoed by the increasing number of clinical trials assessing innovative molecular therapies in the field of EB. Despite remarkable progress, gene-corrective modalities, aimed at sustained or permanent restoration of functional protein expression, still await broad clinical availability. This also reflects the methodological and technological shortcomings of current strategies, including the translatability of certain methodologies beyond preclinical models as well as the safe, specific, efficient, feasible, sustained and cost-effective delivery of therapeutic/corrective information to target cells. This review gives an updated overview on status, prospects, challenges and limitations of current gene-targeted therapies.

Indexed as

Antisense oligonucleotidesEpidermolysis bullosaGene editingGene replacementGene therapyReadthroughsiRNATrans-splicing

Identifiers

PMID34110606
PMCPMC8322229
OpenAlexW3172575232

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.