Evidence map›Paper›PMID 33657662›Full record

ReviewExperimental dermatology2021

Ex vivo gene modification therapy for genetic skin diseases-recent advances in gene modification technologies and delivery.

Vignesh Jayarajan, Evangelia Kounatidou, Waseem Qasim, Wei-Li Di

Open access · hybridAbstract readReview
In one paragraph

Review in Experimental dermatology, 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
1.3field-weighted citation impact, top 20% 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, 23 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Review
  5. Review
  6. Advances in HIV Gene Therapy.International journal of molecular sciences · 2024
    Review
  7. Stem Cell Therapies for Epidermolysis Bullosa Treatment.Bioengineering (Basel, Switzerland) · 2023
    Review
  8. Challenges of Gene Editing Therapies for Genodermatoses.International journal of molecular sciences · 2023
    Review
  9. Article
  10. A Review of Genes Involved in Wound Healing.Medical journal of the Islamic Republic of Iran · 2023
    Review
  11. Article
  12. The Potential of CRISPR-Guided Therapies in the Dermatology Clinic.JID innovations : skin science from molecules to population health · 2022
    Review
  13. Development and clinical translation ofComputational and structural biotechnology journal · 2022
    Review
  14. 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

4 authors at 2 institutions in 1 country.

Vignesh JayarajanInfection, Immunity and Inflammation Research & Teaching Department, Immunobiology Section, UCL Great Ormond Street Institute of Child Health, London, UK.
Evangelia KounatidouInfection, Immunity and Inflammation Research & Teaching Department, Immunobiology Section, UCL Great Ormond Street Institute of Child Health, London, UK.
Waseem QasimInfection, Immunity and Inflammation Research & Teaching Department, Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health, London, UK.
Wei-Li DiInfection, Immunity and Inflammation Research & Teaching Department, Immunobiology Section, UCL Great Ormond Street Institute of Child Health, London, UK.ORCID 0000-0002-4851-1649
University College London · GBGreat Ormond Street Hospital · GB

Funding

Department of HealthMedical Research Council MR/S036989/1
6 · The paper itself

Abstract

Genetic skin diseases, also known as genodermatoses, are inherited disorders affecting skin and constitute a large and heterogeneous group of diseases. While genodermatoses are rare with the prevalence rate of less than 1 in 50,000 - 200,000, they frequently occur at birth or early in life and are generally chronic, severe, and could be life-threatening. The quality of life of patients and their families are severely compromised by the negative psychosocial impact of disease, physical manifestations, and the lack or loss of autonomy. Currently, there are no curative treatments for these conditions. Ex vivo gene modification therapy that involves modification or correction of mutant genes in patients' cells in vitro and then transplanted back to patients to restore functional gene expression has being developed for genodermatoses. In this review, the ex vivo gene modification therapy strategies for genodermatoses are reviewed, focusing on current advances in gene modification and correction in patients' cells and delivery of genetically modified cells to patients with discussions on gene therapy trials which have been performed in this area.

Indexed as

Gene EditingGenetic TherapyHumansKeratinocytesSkin Diseases, Geneticepidermal sheet graftgene editinggenodermatoseskeratinocyte stem cell

Identifiers

PMID33657662
PMCPMC8432139
OpenAlexW3135660229

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.