1. Academic Validation
  2. Application of topical gentamicin-a new era in the treatment of genodermatosis

Application of topical gentamicin-a new era in the treatment of genodermatosis

  • World J Pediatr. 2021 Dec;17(6):568-575. doi: 10.1007/s12519-021-00469-2.
Shan Wang 1 Zhou Yang 1 Ying Liu 1 Mu-Tong Zhao 1 Juan Zhao 1 Huan Zhang 2 Zong-Yang Liu 2 Xiao-Ling Wang 2 Lin Ma # 3 Yong-Hong Yang # 4
Affiliations

Affiliations

  • 1 Department of Dermatology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, No. 56 South Li Shi Road, Xi Cheng District, Beijing, 100045, China.
  • 2 Department of Pharmacy, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, No. 56 South Li Shi Road, Xi Cheng District, Beijing, 100045, China.
  • 3 Department of Dermatology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, No. 56 South Li Shi Road, Xi Cheng District, Beijing, 100045, China. bch_maleen@aliyun.com.
  • 4 China National Clinical Research Center for Respiratory Diseases, Department of Respiratory Medicine, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, No. 56 South Li Shi Road, Xi Cheng District, Beijing, 100045, China.
  • # Contributed equally.
Abstract

Background: The clinical use of gentamicin always lies in its antimicrobial activity in the past as an Aminoglycoside antibiotic. However, in the past decade, there were considerable interests in therapeutic approaches in treating hereditary diseases. Some of the genodermatosis is caused by nonsense mutations that create premature termination codons and lead to the production of truncated or non-functional proteins. Gentamicin could induce readthrough of nonsense mutations and enable the synthesis of full-length proteins. We focus on previous publications on topical application of gentamicin and review its utility in genetic skin diseases.

Data sources: We search the MEDLINE through PubMed, EMBASE databases, and the Clinical Trials Registry Platform from January 1960 to July 2020 using the key search terms "gentamicin, topical gentamicin, genodermatosis, genetic skin diseases".

Results: The application of gentamicin in genodermatosis yielded promising results, both in vivo and in vitro, including Nagashima-type palmoplantar keratosis, epidermolysis bullosa, Hailey-Hailey disease, hereditary hypotrichosis simplex of the scalp, etc. CONCLUSIONS: Topical gentamicin is a potential treatment option for genodermatosis caused by nonsense mutation.

Keywords

Epidermolysis bullosa; Gentamicin; Nagashima-type palmoplantar keratosis; Nonsense mutation; Readthrough.

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