Advances in gene therapy and their application to skin diseases: A review. Issue 1 (July 2021)

• Record Type: Journal Article
• Authors: Shinkuma, Satoru
• Is Part Of: Journal of dermatological science. Volume 103:Issue 1(2021)
• Page Start: 2
• Page End: 9
• Publication Date: 2021-07
• Subjects: αGLA α-galactosidase A -- AAV adeno-associated virus -- ASO antisense oligonucleotide -- COL7A1 collagen type VII α-1 chain -- CBCL cutaneous B-cell lymphoma -- CR complete response -- CRISPR/Cas9 clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 -- CTCL cutaneous T cell lymphoma -- DEB dystrophic epidermolysis bullosa -- DSB double-strand break -- EB epidermolysis bullosa -- EBGraft gene-corrected autologous skin-equivalent grafts (EB patients) -- gRNA guide RNA -- HIV human immunodeficiency virus -- HR homologous recombination -- HSV herpes simplex virus -- IL2RG interleukin 2 receptor subunit γ -- JEB junctional epidermolysis bullosa -- LAMB3 laminin subunit β3 -- LTR long terminal repeat -- MLV Moloney murine leukaemia virus -- NHEJ non-homologous end joining -- NS Netherton syndrome -- PR partial response -- RDEB autosomal recessive dystrophic epidermolysis bullosa -- SCID severe combined immunodeficiency -- SIN self-inactivating -- SPINK5 serine peptidase inhibitor Kazal-type 5 -- T-VEC talimogene laherparepvec -- TALEN transcription activator-like effector nuclease -- ZFN zinc finger nuclease; Epidermolysis bullosa -- Genome editing -- Retroviral vector -- Lentiviral vector -- Adeno-associated viral vector -- Gene therapy; Dermatology -- Periodicals; Skin Diseases -- Periodicals; Dermatologie -- Périodiques; 616.5005
• DOI: 10.1016/j.jdermsci.2021.05.004
• Languages: English
• ISSNs: 0923-1811

Abstract

Highlights: Gene therapy represents a viable treatment option for various diseases. Clinical trials have been conducted on gene therapy addressing genodermatoses. This review summarizes the current use of gene therapy in the dermatological field. Superficial gene-therapy sites can be evaluated for presence of neoplasms. Therefore, dermatologists have an important role in gene therapy application. Abstract: 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.