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Advances in gene therapy and their application to skin diseases: A review

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JOURNAL OF DERMATOLOGICAL SCIENCE
卷 103, 期 1, 页码 2-9

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ELSEVIER IRELAND LTD
DOI: 10.1016/j.jdermsci.2021.05.004

关键词

Epidermolysis bullosa; Genome editing; Retroviral vector; Lentiviral vector; Adeno-associated viral vector; Gene therapy

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Gene therapy is now considered as a treatment for both genetic and acquired diseases, with clinical trials being conducted in the field of dermatology. Different gene-transfer techniques are being used to treat various skin diseases, showing promising results in the current status of gene therapy.
With recent advances in genetic engineering technology, gene therapy is now being considered as a treatment not only for congenital diseases but also acquired diseases, such as cancer. Gene therapeutic agents for hereditary immune disorders, haemophilia, retinal diseases, neurodegenerative diseases, and lymphoma have been approved in the United States and Europe. In the field of dermatology, clinical trials of gene therapy have been conducted, because the skin is an easily accessible organ that represents an attractive tissue for gene therapy. In recent years, gene therapy has been attempted for a variety of skin diseases, such as genodermatoses (including epidermolysis bullosa and Netherton syndrome), cutaneous lymphoma, and malignant melanoma. As a result, it is difficult to grasp the current status of gene therapy in dermatology. This review focuses on each of the gene-transfer techniques currently in use and describes the current status of gene therapy for skin diseases using each technology. (c) 2021 Japanese Society for Investigative Dermatology. Published by Elsevier B.V. All rights reserved.

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