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

Jayarajan, V; Kounatidou, E; Qasim, W; Di, W-L; (2021) Ex vivo gene modification therapy for genetic skin diseases—recent advances in gene modification technologies and delivery. Experimental Dermatology , 30 (7) pp. 887-896. 10.1111/exd.14314. Green open access

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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.

Type: Article
Title: Ex vivo gene modification therapy for genetic skin diseases—recent advances in gene modification technologies and delivery
Location: Denmark
Open access status: An open access version is available from UCL Discovery
DOI: 10.1111/exd.14314
Publisher version: http://dx.doi.org/10.1111/exd.14314
Language: English
Additional information: Copyright © 2021 The Authors. Experimental Dermatology published by John Wiley & Sons Ltd. This is an open access article under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/), which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
Keywords: epidermal sheet graft, gene editing, genodermatoses, keratinocyte stem cell
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10123318
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